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A Report on MyelomaPatient Perspectives

A multi-stakeholder report and recommendations on the role of patients in myeloma research and treatment and the policy framework

in which these take place.

Summer 2013

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MPE Report on Myeloma Patient Perspectives 3

Contents

Foreword 4

Key recommendations 5

Executive summary 7

Methodology 10

Myeloma and patients’ needs

1. Setting the scene: About myeloma 11

2. Best practices in the treatment and care of myeloma – are they being achieved? 23

3. Building a successful partnership with healthcare professionals 33

4. Patients’ needs should be at the centre of research 39

5. An effective policy framework for treatment and care in myeloma 49

6. The power of patient advocacy in myeloma: making it happen 59

Achieving excellence in myeloma care – case studies 69

Acknowledgements 71

About MPE 72

References 73

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4 MPE Report on Myeloma Patient Perspectives

Foreword

This report on Myeloma Patients Perspectives, developed by Myeloma Patients Europe incollaboration with a group of prominent European myeloma experts, deserves wide attention,not only from the myeloma community. It deserves attention from anyone who recognises thefundamental change that is occurring in our healthcare systems and their inevitable evolutiontowards patient-centred care and decision-making.

The report explores patient perspectives on current approaches to myeloma treatment andcare across Europe and their role in future research. It raises questions about how far currentoptions meet patients’ needs and what the barriers and challenges are that limit access to thebest possible treatment and care. It makes recommendations in five critical areas on what canbe done to improve access, better involve patients in research and meet their needs moreeffectively.

Most importantly, this report sets out key recommendations for patient involvement indecisions that affect them directly. It calls for ensuring that patients are equal partners inresearch and are given a formal role in the creation of policy that affects them.

In fact, it goes one step further, to show the need for new models of collaboration between thepatient community, governments, regulators, health technology assessment bodies, healthcareprofessionals, academia and the pharmaceutical industry to drive improved outcomes inmyeloma.

In the last 10 years the survival rates for this rare bone marrow cancer have approximatelydoubled. Yet, when patients are given a diagnosis of myeloma, they face an unknown numberof years of treatment, remission, relapse and uncertainty as their cancer progresses. Many ofthem are diagnosed late and/or still do not have access to the best available treatment and thehighest standards of care.

This report represents the voice of myeloma patients in Europe and a broad consensus onwhat the priorities in myeloma should be to change the status quo. It is a much-neededroadmap for myeloma patient advocacy in Europe.

For its readers, whether coming from government, health service, research or industry, thisreport should become a reference which cannot be ignored in any discussions that concernmyeloma today and in the future.

Dr Faith Daviesmember of the Faculty at the Institute of Cancer Research (ICR), London and ConsultantHaematologist at The Royal Marsden, London.



Key recommendations

Myeloma Patients Europe and all those who endorse the recommendations in this reportshould put in place plans of action with national governments, healthcare professionals,researchers, academia and pharmaceutical industry in order to address the five areas criticalto achieving better outcomes for myeloma patients across Europe:

1) Define best practice in the treatment and care of myeloma

• Work to develop uniform European guidelines for the treatment of myeloma and definingbest practice standards for holistic care including medical and psychological support

• Raise awareness of the symptoms of myeloma among general practitioners (GPs) andorthopaedic specialists to drive earlier referral and diagnosis

• Increase knowledge and capacity of haematologists and other members of themultidisciplinary team to help patients develop strategies for coping with thecomplications, side-effects of treatments and psychological impact of myeloma

2) Build a successful partnership with healthcare professionals

• Encourage patients to develop the knowledge and confidence to work in partnershipwith their doctors in deciding on treatment and to challenge recommendations and seeksecond opinions

• Support improvements in doctors’ training to include better communication skills andattention to the emotional as well as the physical needs of patients

3) Put patients’ needs at the centre of research

• Prioritise clinical trials that are more likely to result in improvements in patient outcomesin the clinic

• Support and invest in collaborative research e.g. through joint projects, EU-fundedresearch consortia and undertakings such as the EU’s Innovative Medicines Initiative

• Make use of the insight of patients in the concept, design and endpoints of clinical trialsas well as the adoption and diffusion of research results

4) Ensure an effective policy framework for treatment and care in myeloma

• Develop national myeloma plans (at least within national cancer plans or national raredisease plans) and within them, clear objectives for diagnosis, treatment andmanagement of myeloma

• Establish national Centres of Expertise and European Reference networks to ensureprovision of access to high quality specialist care for all myeloma patients

• Examine European and national criteria for pricing and reimbursement of newtreatments and to influence this where required to ensure that access is not restricted orprevented on the basis of cost

5) Utilise the power of patient advocacy

• Identify, inform and collaborate with key opinion leaders who can influence and make adifference to the services available to myeloma patients

• Encourage and train individual patients who have the skills to become expert patientadvocates and actively contribute in committees responsible for clinical guidelines,clinical trial design, reimbursement and health policy decisions



The recommendations made in this report are endorsed by:

Amen Israeli Myeloma Patient Foundation (Amen)

Arbeitsgemeinschaft Plasmozytom/Multples Myelom (APMM)

Asociación Española de Afectados por Linfoma, Mieloma y Leucemia (AEAL)

Associação Portuguesa Leucemias e Linfomas (APLL)

Association of myeloma patients of Serbia (AMPS)

Cancer and Stemcell Organization -Coordination Association (KOKDER)

Contactgroep Myeloom en Waldenström Patiënten (CMWP)

Contactgroep Myeloom en Waldenström Patiënten Vlaanderen vzw (CMP vzw)

Društvo bolnikov z limfomom (DBL)

Ege Uni tıp fak

Suomen syöpäpotilaat-myelooma potilasverkosto

Foundation for the Advancement of Bone Marrow Transplantation Switzerland (SFK)

Leukaemiehilfe RHEIN-MAIN e.V. (LHRM)

Multiples Myelom Selbsthilfe Oesterreich (MMSOe)

Myelom Kontaktgruppe Schweiz (MKgS)

Myeloma Euronet Romania (MER)

Myeloma UK (MUK)

MyMu Wallonie-Bruxelles (MyMu)

Slovak Myeloma Society (SMyS-KP)

Slovensko Združenje Bolnikov Z Limfomom In Levkemijo L&L

Society for Assistance to Patients with Oncohaematological Diseases

Udruga Za Podršku Oboljelima Od Multiplog Mijeloma (Mijelom CRO)

Centro de Histocompatibilidade do Norte (CHN)

Klub pacientů mnohočetný myelom (KPMM)



Executive summary

This report has been developed to bring together the many complex issues faced by myelomapatients. Each chapter builds arguments which support the key recommendations to inform anagenda for myeloma patient groups and bring about the change required in their countries aswell as across Europe towards improving outcomes in myeloma.

The challenge of myeloma

Myeloma is a rare type of cancer arising from plasma cells which are found in the bone marrow.It is believed by the Orphanet Europe-wide expert consortium to affect less than two people inevery 10,0001; but this may be a significant underestimate due to variation in data collectionmethods. Its treatment has advanced greatly in the last five years, but the most up-to-datetreatments are not available across all of Europe. Myeloma patients suffer bone pain, anaemia and fatigue, plus increased risk from infection, aswell as immense emotional challenges. Within a relatively short timeframe, many decisions willbe needed about which treatment option to pursue. Some may want the most aggressivetreatment to beat their myeloma, and others will prefer less aggressive treatment that has thepotential to provide a better quality of life. Therefore it is vital that the healthcare afforded tomyeloma patients addresses and meets these very individual needs.The patient’s journey through myeloma meets repeated challenges:

• Rapid diagnosis is essential – but rarely happens, because GPs so rarely encounterpatients with myeloma. Many patients wait up to two years for a correct diagnosis

• Typical progression develops through several cycles of treatment, periods of remissionand relapse: with continual uncertainty and anxiety

• Universal access to the most up-to-date treatments would be ideal, but evidencesuggests that particularly in Eastern Europe, new medications are not available becauseof national reimbursement restrictions. This can even prevent access to medicines thathave been approved by the European Medicines Agency (EMA), which should thereforebe authorised in all EU member states. In non-EU countries, patients cannot even benefitfrom the EU legislation that provides reimbursement when accessing healthcare inanother EU member state

• Many patients, again particularly in Eastern Europe, have no chance of participating inthe clinical trials that could offer them improved treatment or reduced side-effects

• A good quality of life – the aim for most patients – depends on optimal pain relief, fatiguemanagement, and treatments offering longer remissions

• Many treatments involve significant unpleasant side-effects, contributing to a greatlyreduced quality of life and often cause patients to give up their treatment before achievingoptimal results. Resistance also develops in myeloma cells. Both factors mean that newoptions are needed

• Psychological and emotional support is needed as a structured component of care, butacross Europe, patients report that it is not available. This is especially important whenmyeloma returns even though it has responded to a prior treatment (relapse) or wheremyeloma has failed to respond to previous or current treatment (refractory myeloma)

• Palliative care is an important part of treatment and care and should be available fromdiagnosis and not just be considered in the last days of life

• Towards the last days of life, the needs of patients for effective pain relief and of patients,their families and carers for sensitive emotional support, are at their peak



Significant barriers to better treatment and care

As well as the problems already mentioned, economic constraints, regional differences andthe lack of a uniform diagnosis and treatment pathway prevent access to optimal treatmentand care. An important barrier is the inadequate transposition of relevant EU legislation; forexample variation in implementation of the EU directive on cross-border healthcare maydisadvantage patients with less economic resources.

Effective communication between patient and doctor is vital, agreeing realistic, short-termgoals that are constantly reassessed, and taking the emotional stresses into account. Thepatient’s first need is for information, but it must be appropriate and offered at the right time forthe individual. However, communication skills have rarely been taught in traditional medicaltraining, so doctors’ ability varies considerably and would benefit significantly from greateruptake of continuing medical education in all countries. Ideally, training on communicationskills should be added to the curricula of undergraduate medical schools.

For their part, patients need to become more empowered; to develop the confidence andknowledge to seek a second opinion or to be referred to a specialist treatment centre.Myeloma is so rarely seen in primary care that it is important that patients take hold of allavailable information and the support of patient organisations, so that they can take an activepart in managing their own myeloma. More traditional cultures, in which patients tend toaccept their doctors’ opinion without question, may contribute to lower levels of myelomadiagnosis in the newer EU member states.

Patients’ role in research

Patients need information about current clinical trials and the opportunity to participate in themas appropriate.

Continuing research into myeloma is urgently needed, as patients are all working on a limitedtimeframe. As long-term survival is limited to a small number of usually younger patients andto minimise the impact of treatment side-effects, it is particularly important that the advances ofmyeloma research in recent years are rapidly translated into treatments. However, thisresearch is still fragmented and in need of greater coordination, in order to reduce duplicationand enable faster progress. A greater focus on patient needs would bring faster benefit, forexample identifying alternatives to current treatments that cause the worst side-effects.

At this time of economic restraint, patient organisations can help towards this ideal byidentifying priorities and objectives and by making the case for adequate, prioritised fundingfor research studies in both the private and public sectors.

They should also highlight to government agencies the most promising outcomes from clinicaltrials, so that findings on new treatments can rapidly be built into treatment guidelines, whichare many and vary significantly throughout Europe.

Patients and their representatives also have a valuable contribution to make to the ethicalcommittees and the protocol developers for clinical trials and the procedures relating toproduct market authorisation.



Myeloma treatment and care plans in Europe

Most European Union member states have national cancer plans and the EU hasrecommended national plans for orphan diseases (which include myeloma) by 2013. Memberstates have been urged to support national or regional centres of expertise and referencenetworks in order to improve universal access to diagnosis and high-quality healthcare to allpatients with conditions requiring a particular concentration of resources or expertise. Thisobjective has been reinforced by the EU directive on patients’ rights to cross-borderhealthcare. The EU also has recommended improved access to orphan drugs, includingcompassionate provision even before authorisation or reimbursement, and possible incentivesto promote research into these drugs and rare diseases.

Clinical guidelines for diagnosis, management and treatment of medical conditions are basedon the best available evidence, but standards of care do vary in practice because of the rarityof the condition and differences between the various sets of guidelines. Development ofuniform European treatment guidelines would enable myeloma patients to receive the samehigh level of care throughout Europe.

Further policy objectives that could benefit myeloma patients are support for continuingmedical education and reminders about myeloma, so that GPs do not miss the rare myelomadiagnoses; and wider opportunities for patients to participate in ethical and regulatorycommittees for trials and market authorisation.

So the broad framework to improve access to better care in the European Union has been set.There is a great need and opportunity for patient organisations throughout Europe to raise thelevel of specific awareness of myeloma among policy-makers and healthcare professionals.

The role of patient advocacy

Patient organisations have already made a great contribution to supporting and informingmyeloma patients and advocating for priority issues. The current constraints they face are theincreasing patient expectations at the time of economic restrictions and awareness of the needfor a volunteer workforce to provide professional services. There is a significant need todevelop the knowledge and skills of staff and volunteers, including training in advocacy anddevelopment of more expert patient advocates. More strategic partnerships need to be builtbetween patient organisations and other stakeholders involved in myeloma from scientificsocieties to government and industry.

Myeloma Patients Europe will work with the support of its member organisations acrossEurope and other groups which endorse the recommendations set out in this report. Ourobjective is to provide the myeloma community with the educational resources needed in orderto foster more effective advocacy and raise the potential contribution of patient perspectives indecisions concerning myeloma.



Methodology

The information in the report has been compiled from a review of the literature and theinitiatives of regulatory authorities, medical professional bodies and patient organisations,coupled with a series of interviews with myeloma patients.

The report does not provide a country-by-country comparison and has not involved acomprehensive survey of patient views. It is intended to be a starting point for future work forMyeloma Patients Europe and its members.

The report has been prepared with input and consultation from an MPE Writing Group andwider Advisory Board made up of healthcare professionals of all disciplines, researchers,patients and patient advocates.

Its conclusions are therefore fully informed and jointly owned.



1. Setting the scene: About myeloma



Myeloma and patients’ needs

What is myeloma?

Myeloma, also known as multiple myeloma, is a type of bone marrow cancer arising fromplasma cells,which are normally found in the bone marrow. Plasma cells form part of theimmune system.

Normal plasma cells produce antibodies (also called immunoglobulins) to help fight infection.In myeloma, the abnormal plasma cells release only one type of antibody, known asparaprotein or M-protein, which has no useful function. It is often through the measurement ofthis paraprotein that myeloma is diagnosed and monitored.

Bone marrow is the ‘spongy’ material found in the centre of larger bones in the body. As wellas being home to plasma cells, the bone marrow is the centre of blood cell production (redblood cells, white blood cells and platelets).

In myeloma, the DNA of a plasma cell is damaged causing it to become malignant orcancerous. These abnormal plasma cells are known as myeloma cells. Unlike many cancers,myeloma does not exist as a lump or tumour. Instead, the myeloma cells divide and expandwithin the bone marrow.

Myeloma affects multiple places in the body where bone marrow is normally active in an adult,i.e. within the bones of the spine, skull, pelvis, the rib cage and the areas around the shouldersand hips. The extremities – the hands, feet and lower arm/leg regions – are usually notaffected and the function of these critical areas is usually fully retained.

Key points

• There is no cure for myeloma• The goal of treatment is to halt progression, induce remissions and improve quality

of life• It is a very individual cancer and therefore complications and responses to treatment

vary greatly from patient to patient, making myeloma difficult to treat• Multidisciplinary teams are needed to manage myeloma effectively• Diagnosis and treatment bring profound emotional challenges to patients• Treatments have improved dramatically in the last five to 10 years; however

myeloma outcomes are still relatively modest – much more needs to be done• The cost of treating myeloma has increased dramatically over the last 10 years

and this is likely to continue

1. Setting the scene: About myelomaMyeloma – a rare form of bone marrow cancer – is little known among the public and a GP islikely to meet only one or two patients in their career. This first chapter examines the nature ofthis rare cancer, what causes it and how it is treated. Most importantly, it looks at the manycomplex physical and emotional effects that myeloma has on patients – and what treatment,support and other initiatives would help them best to cope with this devastating cancer.



Most of the medical problems related to myeloma are caused by the build-up of myeloma cellsin the bone marrow and the presence of the paraprotein in the blood or in the urine. Commonproblems are bone pain, bone fractures, tiredness (due to anaemia), frequent or recurrentinfections (such as chest and bladder infections), kidney damage and hypercalcaemia (raisedcalcium level in the blood).

Some people develop myeloma after having been diagnosed with a condition called MGUS(Monoclonal Gammopathy of Undetermined Significance). This term describes the condition ofthe raised abnormal protein seen in myeloma (the paraprotein), but where there are no otherfeatures of myeloma (less than 10% plasma cells in bone marrow and no evidence ofmyeloma bone disease). In MGUS the M-protein level is less than 30g/L.

The risk of transition from MGUS to active myeloma is very low; only a 1% chance each yearof follow-up. Even if the myeloma cells are at a higher level of 10 – 30% of the total bonemarrow, and the M-protein level may be higher than 30g/L, the growth rate can be very slowand with no symptoms. This is normally referred to as smouldering myeloma.

Both MGUS and smouldering myeloma can change very slowly over a period of years and donot require active treatment. It is very important to establish the correct diagnosis,distinguishing MGUS and smouldering myeloma from active or symptomatic myeloma, whichdoes require treatment.

A very small proportion of myeloma patients (1 – 4%) can progress to plasma cell leukaemia,which is characterised by high levels of plasma cells circulating in the blood2 and shortersurvival.

Myeloma quick facts

• Myeloma is rare, representing about 1% of all cancers• It affects over 84,000 people in Europe• A type of bone marrow cancer, myeloma arises from plasma cells in the

bone marrow• Affects multiple places in the body where bone marrow is active• More common in people of African origin• Affects more men than women• Most commonly affects people over 60• Often described as the ‘unknown’ cancer, there is no proven cause• Most common symptoms and side-effects are bone pain, bone fractures,

tiredness, frequent or recurring infections, kidney damage and hypercalcaemia• One quarter of patients diagnosed with myeloma will die within one year• Myeloma will always relapse – there is no cure



What causes myeloma?

The cause of myeloma is believed to be a combination of genetic and environmental factors.Although a large amount of research has investigated the potential causes of myeloma, nothinghas been proven to date. The factors most commonly implicated in the onset of myeloma arethought to be:

• The ageing process: As myeloma is more common later in life, it is thought thatsusceptibility may increase and effective immune function reduce with age, or thatmyeloma may result from a lifelong accumulation of toxic insults or antigenic challenges

• Exposure to certain chemicals, radiation and a weakened immune system: It is likely thatmyeloma develops when a susceptible individual has been exposed to one or more ofthese potential causal or trigger factors

• Being overweight or obese may also increase the risk, although more research is needed3

• Genetics. There is a rare tendency for myeloma to occur in families, but the likelihood isvery low and no tests are currently available for this. Even when myeloma occurs morethan once within a family, it may be due to a common exposure to environmental factors

How does myeloma impact patients?

Myeloma is a complex cancer that impacts patients both physically and emotionally. This non-exhaustive list shows the most common types of physical and emotional impact.

Physical:• Fatigue. Usually caused by anaemia, fatigue results in reduced independence, reduced

ability to work and financial difficulties• Pain

• Fractures and breaks

• Mobility problems

• Infertility. This can be a problem for younger patients, particularly now that newtreatments can often prolong lives for more than five years

• Risk of infection. The continuing need to reduce the risk of day-to-day infection, becauseof a weakened immune system, often causes problems with maintaining an active sociallife

Emotional/Psychological:• Anxiety and depression. A feeling of stigma, a sense of isolation and fear of eventual

relapse all contribute to anxiety and depression• Profound emotional impact. As well as being an incurable cancer, myeloma is very rare

and described as an ‘unknown cancer’, further adding to the emotional burden• Decreased libido. Some treatments have a negative impact on sexual health and libido,

which are often overlooked between patient and doctor, but can contribute to depressionExclusion and loss of self-esteem:

• Friends can be lost if they are unable to understand the situation • Hair loss from chemotherapy can heighten the feeling of exclusion • Living with the knowledge of myeloma can also bring a sense of personal loss – of time

and opportunity• These issues all contribute to a loss of self-esteem



Judy’s story – The impact of myeloma

When Judy was diagnosed, she was 33, with two small children and a full-time job as astockbroker. She was used to working very hard, but began to catch a lot of infections.Blood tests and a bone marrow biopsy revealed smouldering myeloma.

She was monitored for five years and gave up her job when her doctors felt her risingparaprotein level and low haemoglobin were indicative of progression to active myelomaand would soon affect her quality of life. She required a stem cell transplant, which gave agood partial remission.

Her paraprotein started creeping up after four years of remission from the transplant, butJudy had a suboptimal response to subsequent treatment. After a further year and a halfshe began to feel pain in her hip, which intensified over the coming months and an MRIshowed significant bone involvement from the myeloma. A second successful transplanthas allowed Judy to get on with her life to a large degree. Her immune system is the mainproblem; it was affected by myeloma and worsened after treatment, so she has to beextremely careful to avoid travelling by plane and going to busy places where she could beat risk of infection.

Myeloma and dealing with the drugs has also meant a huge mental and emotional strain.“It’s like living on the edge of a cliff most of the time. Even if you are a ‘glass half-full’ sort of person it can be quite wearing, not knowing when the next relapse will come, andwhether the drugs will be available in your country. With some cancers, after five years youare considered to be cured; with myeloma it’s the opposite; you know it’s always going torelapse.” Judy also describes feelings of guilt for the anxiety her illness has given herfamily.

Judy feels she was lucky in how quickly her myeloma was diagnosed, but that also gaveher a long period of uncertainty while her smouldering myeloma was not treated. She isglad she has had access to good information and support. However, she feels that fewconsultants have a full understanding of what treatments would be best for an individualpatient; not always taking into account whether a patient is at high risk or low risk, orrunning out of treatment options. And every centre with a myeloma service, she feels,should be able to advise patients on what clinical trials are on offer.

Judy Dewinter,

Myeloma patient, UK

“It’s like living on the edge of a cliff most of thetime. Even if you are a ‘glass half-full’ sort ofperson it can be quite wearing, not knowingwhen the next relapse will come, and whetherthe drugs will be available in your country.”Judy



Do many people get it?

Myeloma is a relatively rare cancer:

• Myeloma is rare†, representing about 1% of all cancers in white people6 and 15% ofblood cancers7

• It is the second most common blood cancer after non-Hodgkin’s lymphoma. • Myeloma is more common in people of African origin and less in Asians6,8

• It affects more men than women and most commonly affects people aged over 60In 2008 the European Union’s 27 member states were estimated to have about 73,000myeloma patients and the whole of Europe over 84,0009‡. The exact number of myelomapatients may be understated, as numbers are only derived from numbers of patients survivingfor five years. Just under 37,000 new patients were diagnosed in 2008 alone9.

In Europe as a whole, a higher proportion of the population has myeloma in WesternEuropean countries than in Eastern Europe. This finding may at least in part due to lesseffective diagnosis or different methods of data collection.

Source: International Agency for Research on Cancer: Globocan 20089

* the annual number of new cases multiplied by the probability of survival for 5 years

Estimated 5-year prevalence per 100,000 people*

† A rare disease is defined as one affecting not more than 5 per 10,000 people (European Community Action programmeon rare diseases 1999-2003); other world regions have slightly differing definitions‡ Derived from the latest cumulative estimates of 5-year surviving patients, from Globocan 2008, International Agency forResearch on Cancer. The data for the whole of Europe represents EU-27 plus Belarus, Croatia, Moldova, the RussianFederation, Ukraine, Iceland, Norway, Albania, Bosnia Herzegovina, Croatia, Macedonia, Montenegro, Serbia andSwitzerland.



Some countries within the European Union either have more people with myeloma in relationto the size of their population, or are diagnosing them more effectively. The number of newcases diagnosed varies by well over three times between the 27 countries.

For both incidence and 5-year survival, the same ten countries rank highest and survival ratesin Luxembourg, Italy, France and Belgium are rather better than in the other countries. Withthe exception of Slovenia, lower rates of both incidence and surviving patients are shown bythe Eastern European member states.

Other sources indicate that the 5-year survival rates vary from 23.1% to 46.7% of myelomapatients across the European countries3. All of these observations should be used withcaution, as they may be affected by differing reporting methods. It is likely that in countrieswith very high incidence rates, some patients with MGUS may be diagnosed as havingmyeloma; this could also explain their high survival rates and consequently a low incidencemay mean missed diagnosis and/or poor survival.

Patients with MGUS may be diagnosed as having myeloma; this could also explain their highsurvival rates and consequently a low incidence may mean missed diagnosis and/or poorsurvival.

Sources: International Agency for Research on Cancer: Globocan 20089 / UN World Population Prospects 200810

However, the best rates of survival after five years in those countries are more than 4.5times as great as the lowest rates.


Myeloma incidence in EU-27, 2008: New cases per 100,000 population

Myeloma prevalence in EU-27, 2008: 5 year surviving patients per 100,000 population

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In the European countries that are not (or not yet§) members of the EU, several features are clear:

• With the exception of Norway, Iceland, Switzerland and Croatia, recorded incidence isvery significantly lower than in most EU member states

• The lowest level of recorded incidence (in Moldova and Albania) is only 16% of thehighest non-EU country (Norway) and 13% of the highest EU member state

• Combining both sets of data suggests that a significantly lower level of diagnosis isachieved in most of the non-EU member states and in the Eastern EU member statesapart from Slovenia

• 5-year survival rates for Iceland, Norway, Switzerland and Croatia are again significantlybetter than in the other non-EU countries and as for their higher recorded incidence rates,these four countries rival the better-performing western EU member states

As for other European countries, the incidence of myeloma in Turkey, for example, is7.1/100.000 for males and 4.5/100.000 for females. However, these data are not official andshould be interpreted with caution**.


§ Croatia was due to join the EU on 1 July 2013** Source: KANKO-Cancer Coalition of TURKEY (report 2013)

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How is myeloma treated?

Although myeloma cannot be cured, it is highly treatable in the vast majority of cases and thetreatments available have made very significant advances in the last ten years. A wide varietyof drugs and combinations is now available and the exact treatment plan should be decidedfor each individual.

Aims of treatment:

Following diagnosis, an initial course of treatment is agreed with the aim of reducing the levelof myeloma cells, relieving the symptoms and complications, improving the patient’s quality oflife and the length of survival7. After a period of remission, many myeloma patients suffer arelapse and the treatment may then be repeated or an alternative combination given. Patientsoften experience several periods of remission followed by relapse. Ultimately no furthertreatment yet available can stop the course of the cancer.

Treatment of myeloma

• Treatment decisions are made on the basis of:

o Stage and risk of myelomao Individual patient’s health and preferenceso A balance of the benefits and side-effects of treatmento Prolonging lifeo Achieving the best quality of life for the patiento Logistics of available treatments.

• Treatment and management of myeloma is complicated and requires a multidisciplinaryteam, including orthopaedic, renal and pain specialists

• Choices for one patient may not suit another

• Choices are supported by clinical treatment guidelines from national health authorities orprofessional medical bodies and complement the legal framework provided by the EMAand the reimbursement decisions of national pricing authorities

• Many sets of guidelines and differences in the availability of medicines results insignificant differences in treatment across Europe

Available treatments:

People given a diagnosis of myeloma will be treated according to the nature and stage of the disease, their age, fitness and preferences. Those diagnosed with MGUS or smoulderingmyeloma will not need treatment. Broadly, younger and/or fitter patients are first likely to beoffered a combination of drugs, followed by intensive treatment. This consists of high-dosechemotherapy to destroy the myeloma cells, followed by replacement of healthy stem cells via a stem cell transplant from the patient or a tissue-matched donor.

Older and/or less fit patients (over about 70 years) or those who are less able to tolerate theside-effects of intensive treatment will be offered a similar (although often with attenuateddoses) combination of drugs, but no high-dose chemotherapy and stem cell transplant.

A typical drug combination will most probably include:

• A chemotherapy drug to kill the myeloma cells (cyclophosphamide, melphalan ordoxorubicin)

• A steroid to suppress inflammation (dexamethasone or prednisolone)



• Other newer anti-myeloma treatments including thalidomide (ThalidomideCelgene®),lenalidomide (Revlimid®) and bortezomib (Velcade®). These can improve theperformance of chemotherapy drugs when given in combination11, and prevent myelomacells from surviving

• Pain relief as needed; usually non-steroidal anti-inflammatory drugs (NSAIDs) or, in thelater stages, morphine

Radiotherapy is sometimes the first treatment given to specific areas damaged by myeloma,as it can relieve pain more quickly than chemotherapy or other painkillers. It damagesmyeloma cells but also normal plasma cells, so it is only used to treat small areas that arecausing particular problems12.

As well as treating the myeloma itself, it is important to prevent the myeloma from causingfurther damage to the bones, which is achieved with bisphosphonate treatment - zoledronicacid (Zometa®), pamidronate (Aredia®) or clodronate (Bonefos®, Loron®).

Response to treatment:

If treatment is successful, the number of myeloma cells in the bone marrow will be reduced toa low level, allowing the bone marrow to recover, or even eliminating them. If the myelomaremains stable, it is said to be in remission7.

In order to check the progress of treatment, patients will undergo regular blood and urine teststo determine their paraprotein levels, which should be falling. They should also beexperiencing less bone pain, reduced anaemia and a general improvement in health7.

Choosing between available treatments:

Myeloma specialists will select the most suitable treatment for the individual patient and stageof disease; balancing the benefits and side-effects of the treatment. The aim is to prolong lifeand achieve the best possible quality of life for patients, making full use of a multidisciplinaryteam including orthopaedic and renal specialists and providing adequate control ofcomplications and symptoms.

Treating myeloma involves complicated choices for specialists, GPs and other healthcareprofessionals together with the patient – in terms of the choice of drugs, duration of treatment,monitoring and the type and range of supportive care needed. The choices for one individualpatient may not suit another, because of personal preference, age, genetics, general fitnessand other medical conditions.

Clinical guidelines

Treatment decision-making is often supported and informed by the presence of clinicaltreatment guidelines from national health authorities or professional medical bodies thatrecommend appropriate treatment and care for myeloma patients.

Recommendations contained within guidelines are exactly that: guidance rather than a set ofrules, and are influenced by and must operate alongside the legal framework provided by themarket authorisation of medicines by the European Medicines Agency and the reimbursementdecisions of national pricing authorities.

National guidelines are available for the entire myeloma pathway e.g. those from the UKMyeloma Forum and British Committee for Standards in Haematology or the ItalianHaematological Society, and take account of the medicines available and reimbursed in thatcountry13,14,15,16,17. The US has its own guidelines18,19,20.



Broader international guidelines on specific aspects also come from the International MyelomaWorking Group (IMWG) of the International Myeloma Foundation21 and the European Societyfor Medical Oncology22.

Clinical guidelines are based on the aggregation of best available evidence or, in its absence,on consensus of expert opinion. They are intended to support and guide healthcareprofessionals in their work without replacing their knowledge and skills23. Guidelines shouldideally present the best, most up-to-date recommendations on treatment options based onreview of current validated research24. Their use can preserve professional autonomy andrationalise the increasingly complex sets of evidence into relatively straightforwardrecommendations and also protect doctors against criticism or even legal action25.

However, it is not always possible to account for every type of patient and treatment decisionin myeloma and inevitably, some doctors will always have their own opinion so in practiceguidelines are, often with good reason, not always followed. Together with the many sets ofguidelines and the differences between the medicines available in different countries, thisresults in significant differences in treatment and care across Europe.

Recent advances in research

Many new developments in the treatment and management of myeloma over the last fewyears have had a significant impact on the way it is treated. Research is continuing to developnew treatments and to use existing treatments in a better, more effective way. Treatments formyeloma can be very effective at halting its progress, controlling the symptoms and improvingquality of life.

Use of the recently-developed treatments has made it possible to aim for a completeresponse11 (i.e.control of the myeloma and a stable remission) in a larger proportion of patientsthan previously possible; meaning that myeloma is increasingly considered as a chroniccondition in some patient groups.

Myeloma is a highly complex and continually evolving cancer. At its core are a series ofgenetic changes that interact with each other, causing the myeloma to progress and ultimatelybecome resistant to treatment. Studying the genetic changes that occur in myeloma cells isfundamental to understand the biological and cellular changes that drive the cancer. Mostimportant has been the identification of a number of genetic alterations that influence apatient’s prognosis. Recent advances in cytogenetic and fluorescence techniques and gene-expression profiling are now enabling these genetic alterations to be detected. The next 5 – 10years is likely to see an upsurge in these discoveries being brought into clinical trials, leadingto a clearer understanding of how treatments can be tailored to individual patients.

Is myeloma costly to treat?

Historically, the cost of myeloma treatment in the vast majority of cases was relatively cheap.However, the introduction of new treatments has brought with it a dramatic increase in costsboth in terms of the individual price of drugs as well as how they are used.

As a consequence, the costs of treating myeloma are among the highest of all types ofcancer26 with newer medications like bortezomib (Velcade®) or lenalidomide (Revlimid®)costing several thousand euros per month27. These costs are currently coming under intensescrutiny, especially in terms of whether they deliver value for money.



Summary

Myeloma is a very complex cancer that is not yet fully understood. The available treatmentscan offer substantial benefit, especially the more recently-developed medications that enhancethe performance of chemotherapy drugs when taken in combination with them. Genetictechniques under development also offer significant promise for the future. However, after anumber of cycles of remission and relapse, still ultimately there is no cure, so that patientsface a great deal of uncertainty.

Taking all these different aspects into account, it is therefore important to ensure a holisticapproach to treatment – one that focuses on the person as well as the cancer.

Mika’s story – Still well after 25 years!

Although at age 20 Mika was given three years to live, that is now 25 years ago and todayhe is well, happy and able to do almost everything he wants (except play basketball).Recently he met his first myeloma consultant, who said Mika is still his youngest patient.

As a young man he was in the Finnish army and began to experience pain in his leg whenrunning. Walking down the stairs one day he broke his femur. “My life had not evenstarted,” he says, “when the doctor told me I had cancer.” The break was due to aplasmacytoma. At that time (in the mid-1980s) it was very hard to find information, with nomyeloma associations or internet. Mika took a course for cancer patients, but becausemyeloma was so rare, there was little specific information. He felt it was hard to askquestions of the doctors and nurses.

Mika had surgery to remove the plasmacytoma, followed by radiotherapy; and this wasrepeated in 2000. In 2008 he was given chemotherapy for myeloma and had his stem cellsharvested and frozen for a transplant, but so far that has not been needed. “I know and I hope that some day a transplant will allow me to continue my life; it’s easy forme, knowing that the stem cells are there and I don’t need some from another donor.” Hefeels that his myeloma treatment in Finland has been excellent, but psychological supportis not so readily available. Instead, social and emotional support and the information tohelp patients to cope with everyday life have come mainly from the voluntary cancerassociations. In Finland, the government plan of 2011 includes a national cancer centrewith important functions for the patient associations.

Mika is starting to work full time for the cancer association and considers himself a luckyman. To others with myeloma, he says: “If you know a lot about your disease, it will helpyou a lot. Better understanding of the behaviour of the disease can help manage theuncertainty and alleviate unfounded fears. I think being positive is the only way to live,trying to have a normal life, going to work, and being with my wife and my dear friends.”

Mika PeltovaaraMyeloma patient, Finland

“Better understanding of the behaviour of thedisease can help manage the uncertainty andalleviate unfounded fears.” Mika


2. Best practices in the treatment and care of myeloma – are they being achieved?



People with a complex cancer like myeloma need very specific input from all the differentmedical, scientific and social members who together make up a multi-disciplinary team thatworks in a co-ordinated, holistic way. This chapter looks at what those needs are and revealsmajor shortcomings in many countries of Europe.

Examination of the barriers preventing access to the best standards of care shows latediagnosis, poor access to the most recent treatments, to well-informed medical care, clinicaltrials and psychological support alongside differences due to geographical location, age andsocioeconomics.

2. Best practices in the treatment and care of myeloma – are they being achieved?

Key points

• Rates of survival appear to reflect a variation in diagnosis, treatment and care between European countries

• The main barriers to optimal care are:o Delayed diagnosiso Poor access to authorised treatmentso Waste of resourceso Regional differenceso Variation in reimbursement policies

• Older patients are more likely to have other medical problems in addition to myeloma

• 5-year survival is much greater in younger patients• Poorer outcomes are seen in people with lower incomes, lower levels of education

and by country of residence• Culture may have a bearing on access to treatment and support. Many people find it

harder to question and therefore harder to seek access to more information, latesttreatments, clinical trials and/or psychosocial support.

What should patients expect from their treatment and care?

Although not exhaustive, this list highlights patients’ expectations about what constitutes besttreatment and care.

Up-to-date treatment:Significant advances have been made in the treatment of myeloma over the last ten years,including availability of new newer proteasome inhibitors e.g. carfilzomib andimmunomodulatory drugs, e.g. pomalidomide. These new possibilities promise significantlybetter results than traditional approaches. Given the relapsing and remitting nature ofmyeloma alongside the fact there is currently no cure, patients need access to the besttreatments that research can offer them.



Living with myeloma – I want to know everything about it

“I believe it is important that newly diagnosed patients, still trying to overcome the shock oftheir diagnosis, are put in touch by the hospital with those who have gone through a stemcell transplant and have led a normal life for several years before needing furthertreatment.

“I decided two objectives soon after my myeloma diagnosis twelve years ago – I wasdetermined to see this cancer off, and to study it and understand the nature of the diseaseand its treatments. I knew nothing about haematology but I started to access the latestpublications on the internet and attended many myeloma seminars. The knowledge Iacquired became a powerful attribute to my fight against myeloma, and after years of studyI became accepted by some as an expert patient.”

Stewart McRobert

Myeloma patient, UK

“I was determined to see this cancer off, and to study it and understand the nature of thedisease and its treatment.” Stewart

Information: Initial shock and numbness at the time of diagnosis may be mixed with anger (why me?),denial, or relief at being given a reason for hitherto unexplained symptoms. The first need isfor information, which must be understandable (not too technical) and up-to-date.

Information that applies directly to the individual and to the stage of myeloma enables patientsto understand it more fully, to play an informed part in treatment decisions and to feel more incontrol. However, not every patient wants all possible information; the important thing is thatthey have the level of information that they need28.

Effective communication:As well as information, patients need effective communication with their doctors and enoughtime to digest the options. No action may be needed beyond regular monitoring, but it isimportant that patients understand why that decision is reached, so they do not feel as ifnothing is being done.

Psychosocial support:Patients are vulnerable to depression and should have access to regular assessment andpsychological support or counselling. It is well established that the emotional support ofpartners, family and friends and any other carers is essential to help them to adjust andcope29. Partners and other carers have a crucial role and offer a reason to go on fighting29

(often involving great personal stress). Ideally this should be matched by practical measuresincluding efficient arrangements from hospital authorities to enable regular, stress-freeattendance for treatments and assistance from social services and complete access to allrelevant social security benefits.

Hard evidence is scarce on the availability of psychosocial and emotional support, butanecdotal evidence suggests that it is rare, especially in Eastern Europe.



†† CMWP – Contactgroep Myeloom en Waldenström Patiënten

Carers need support too:The emotional burden on people who care for myeloma patients – often their partner oranother family member – is sometimes even greater than that on the patient5. A recent studyacross four hospitals in the UK showed that almost half of myeloma patients’ partners (48.8%)reported signs of anxiety and 13.6% showed signs of depression5.

Carers have many of the same information needs about myeloma and its treatment aspatients, but also many unique ones that the patient may not share and may not want to knowabout. Healthcare professionals should ensure that carers and family have the support theyneed to manage continuing side-effects or complications, lack of local health services asneeded and the changes that myeloma has caused to the patient5.

Patient organisations are offering a very supportive base for carers in many Europeancountries, especially in providing forums for comparing notes with other carers. TheNetherlands myeloma association††, for example, supplies extensive and diverse informationfor both patients and carers.

Access to expertise within a multidisciplinary team:As well as a specialist haematologist to propose the most appropriate treatment for themyeloma itself and for any orthopaedic or renal complications, patients need the regularsupport of different members of a multidisciplinary care team. Members of this team willcommonly include a specialist myeloma or haematology nurse, a radiotherapist, anorthopaedic surgeon, a renal specialist, a physiotherapist, a dietician and palliative care staff.

Access to a myeloma nurse specialist:Trust and confidence in the team members is essential29 and among them, specialist nursesare especially important. Patients report easier communication with nurses than with theirdoctors, because of less time pressure and a better understanding of the daily impact ofmyeloma. Specialist nurses can also help in practical ways.

Access to treatment when needed:The decision to treat or not to treat immediately is an important one. Treatment, if needed, willdepend on the stage of myeloma, the presence or absence of symptoms or complications andgeneral health. Ideally, it should be tailored to the individual in terms of their age, fitness andother medical conditions. In years to come it may be possible to offer truly personalisedtreatment, taking account of individuals’ genetic makeup (see page 46).

The opportunity to take part in research:Many patients also welcome the chance to participate in clinical trials of new drugs. Theseoffer the dual benefits of possibly being helped by tomorrow’s drugs today and also receivingintensive monitoring while the trial is running.

Shared decision-making:It is important that patients feel their views and preferences are taken fully into account indecisions about treatments. Treatment can take various routes, many of which involve abalance between benefit and side-effects. Effective communication and shared informationbetween doctor and patient should take into account all the medical and psychological factors,so that both can arrive together at the best overall course of action.



The last days of life:The time will come when the myeloma has progressed so far that no treatment will alter itscourse30. At this time, extreme sensitivity is needed on the part of the doctor in making theoutcome clear to the patient. The care pathway should ensure that the patient’s preferencesare being met and that the palliative care team, social services and primary care team aremonitoring the physical and emotional needs of both the patient and their family. Inappropriatetreatments should be stopped and major symptoms, particularly pain, controlled to keep thepatient as comfortable as possible14.

There are a large number of ways and a broad spectrum of how patients come to terms withthe last days of life. For example, while some patients are able to draw comfort from hope,spirituality, family support29. others may become withdrawn, distant and tearful30. Suchreactions may be because they are themselves unable to come to terms with the end of life, orbecause of difficulties in talking openly about it with the family and a wish to protect them.Family members may choose to focus on the present time, rather than the future, because it istoo upsetting30. For both patient and family, emotional support is needed just as much aseffective communication30.

Christa’s story – A positive outlook is everything

It is exactly seven years since Christa was diagnosed with myeloma, after investigation forsevere anaemia. Since then she has endured a succession of treatments that have givenher remission and relapse, over and over again. Her first two treatments were transplantswith her own stem cells, which gave her a partial remission and allowed her to return towork for a year. But when the relapse came, Christa’s doctors were unable to harvest herstem cells. Treatment with thalidomide left her feeling extremely tired and low, so it wasreplaced with Velcade® and a good remission followed.

At the next relapse, Christa agreed to an allogeneic transplant with stem cells from hersister, who fortunately was a good match. The time after the transplant was the hardesttime of all, Christa says; not knowing if the transplant would work, not able to eat, or go outor see people in case of infection. But other things did help – including some homeopathictherapies, yoga, meditation and breathing techniques; also sports when she was wellenough again. “Most importantly, I learnt the cancer cells are not my enemy. I had toconsider them my friends that wanted to tell me something, and I had to find out what itwas. I had to change my personality a bit and try to live with more consciousness as everyday could be the last day. Myeloma cannot be cured so you have to think about that; I reada lot about philosophy and that helped me.”

Christa has worked with the self-help groups of her myeloma patient association for years,since soon after her own diagnosis. Before joining she thought she might hear “too manybad things”, but she found it very helpful to exchange experiences with other patients. Aftera sustaining therapy with lenalidomide, Christa had a relapse with a solid plasmacytoma inthe liver, but this has been eliminated recently by radiotherapy treatment. “It’s a never-ending story,” she says. “Every myeloma case is different. My advice to every patient is –inform yourself.”

Christa Kolbe-Geipert, Myeloma patient, Germany

“Every myeloma case is different. My advice toevery patient is – inform yourself.” Christa



What is lacking in myeloma treatment and care? The patients’ view…

Research has shown that many myeloma patients have a lower quality of life than those withother forms of blood cancer5,31. A recent UK study5 revealed that one quarter of myelomapatients investigated could describe care needs that were not being met, while their carersreported similar care needs and a level of distress even higher than that of the patients.

The needs expressed5 were:

• Health-related, e.g. pain interfering with daily life, reduced physical functioning, fatigue,side-effects

• Emotional/psychological, e.g. anxiety about treatments and side-effects, anxiety aboutthe burden on carers, anxiety about refractory myeloma or relapse, depression

• Practical, e.g. access to hospital parking

• Financial, e.g. access to life insurance

• Social, e.g. a lack of knowledge and understanding about myeloma among the generalpublic

In addition to the needs recorded in that study are the difficulties of achieving shared decision-making between patient and doctor.

What is preventing myeloma patients from receiving the beststandards of treatment and care?

Clearly not everyone with myeloma in Europe is receiving the best possible treatment andcare from the outset. These are the main barriers:

Delayed diagnosis:As myeloma has such a devastating physical impact, early diagnosis is important in order tobenefit from treatment and prolong life of an acceptable quality. In some EU countries, patientscommonly report having waited up to two years before their myeloma was diagnosed. Duringthat time, patients can suffer fatigue, pain and anxiety and a reduced quality of life. GPs andorthopaedic specialists commonly see the symptoms of myeloma, such as back pain, but arecent survey in Belgium found that the a GP is likely to see only one or two myeloma patientsin a 30-year career. Delay means that the patient’s condition is worse at diagnosis and is morelikely to be suffering bone fractures, renal problems or infections that can make deliveringchemotherapy more difficult.

Information is needed to help alert GPs and orthopaedic specialists to the very smallproportion of their back pain sufferers who may have myeloma and to raise awareness ofother symptoms possibly due to myeloma. Ideally people with suspected myeloma shouldbenefit from a rapid-referral system for specialist diagnosis.

Poor access to authorised treatments:Anecdotal evidence shows that treatments approved by the European Medicines Agency(EMA) are often not available equally across Europe or only available with restrictions. Thevariation in treatment within and between different countries results from different doctorpreferences and healthcare systems, level of state or private sector funding and also from thevarying national reimbursement policies.

For example, Revlimid® has received approval for marketing authorisation by EMA as asecond-line treatment (i.e. after the first relapse), in combination with dexamethasone.However in Belgium, it was initially only reimbursed for third-line treatment after relapse while



under treatment with Velcade® and for eight cycles of treatment. Further, if the patient was notin at least partial remission after four cycles of Revlimid®, reimbursement of this medicine wasstopped. The doctors could not prescribe Revlimid® according to the European authorisation.The Belgian patient organisation has worked with doctors and the pharmaceutical company toadvocate for reimbursement criteria that allowed Belgian patients to benefit from the medicine,as recommended by the EMA.

In some Eastern European countries the reimbursement decisions relating to myelomatreatments are described by patient organisations as “catastrophic”. In Hungary, both Velcade®

and its generic equivalent are only reimbursed for three cycles, and Revlimid® is “only adream”. The out-of-pocket cost of Revlimid® for patients themselves, at €6-7,000, is too highfor most patients. France, Germany, Belgium and most other Western European countrieshave more generous reimbursement systems.

Brain drain:The difficulty that doctors face in attempting to provide care in Eastern European countrieswith less advanced treatment options is causing many to leave in search of a more favourableworking environment where they have access to the most up-to-date treatments. For myelomapatients in those countries, this makes the prospects even worse.

Regional differences:The standard of care available varies according to where patients live. University hospitals, forexample, have myeloma specialists who may be engaged in current drug trials and are fullyaware of the latest findings on the uses and advantages of innovative treatments. Incomparison, patients of rural, local hospitals are often in the care of general haematologists oroncologists who may not be fully informed on the latest research developments, clinical trialsand complexities of myeloma.

Geography, age, socioeconomics and culture:Myeloma mainly affects people in the older age groups, with fewer treatment options andaccess to clinical trials. They are also more likely to suffer other medical problems, whichwould weaken their overall condition32. Five-year survival is much greater in youngerpatients32.



Johan’s story – Lucky to have the medical costs covered

Johan lives in Belgium, and has had myeloma for 15 years. Out of the treatments availablein the early years, he was only treated with bisphosphonate. After five years he was givenVAD induction treatment, to be followed by an autologous stem cell transplant, but thetransplant could not proceed as the induction therapy was less than successful.

The myeloma remained stable for seven years, but radiotherapy was needed for twoplasmacytomas and again later for another that was affecting the pelvic bone. A treatmentaccording to the Velcade®-dexamethasone schema (4 cycles) was started, followed bycyclophosphamide, growth hormone treatment and a further stem cell transplant. After allthese different treatments, he went onto consolidation treatment with thalidomide aloneand then in combination with Velcade®-dexamethasone.

“I am very satisfied with the medical care I have received,” says Johan. “All the treatmentshave been undertaken in consultation with my doctor, and an added plus is that through mywork, I have additional insurance for medical expenses. However, the criteria for treatmentof myeloma and the repayment terms of the health insurance are different from country tocountry. ”

Johan CreemersMyeloma patient, Belgium

“the criteria for treatment of myeloma and therepayment terms of the health insurance aredifferent from country to country.” Johan


15 – 49 50 – 59 60 – 69 70 – 79 80 – 99

Five-year relative survival rates by age, England, 2005 – 2009

Source: Cancer Research UK 201232


Within the last ten years, an increased risk of myeloma and poorer outcomes have beenshown in people with lower incomes or a lower level of education, although these differenceswere not detected by earlier studies32,33. Distance to medical treatment may delay or deterrural residents from seeking diagnosis and treatment; also people from lower socioeconomicgroups are more likely to present with more advanced myeloma.

It is possible that those in lower socioeconomic groups may less frequently seek diagnosisand receive less extensive investigation from their doctors33. It has been shown that olderpeople, women and those from socially disadvantaged areas have a lower referral rate for hippain34. This may be related to more difficult access to specialist care, or practices indisadvantaged areas having a higher workload and more patients with a higher number ofcomorbidities34.

Culture may also have a bearing on access to treatment and support. Doctors havetraditionally held a position of knowledge and authority which many people, especially in theolder age groups, those without a strong education and those in the former communistcountries, find difficult to question. It is much harder for these people to seek more information,or access to the latest treatments, clinical trials or psychosocial support.

Unmet psychosocial needs:Myeloma patients are known to suffer pain, fatigue and sleep disturbance; all of which willhave an impact on everyday life. This is frequently linked with anxiety and depression, yet fromthe accounts given by patients, medical care tends to focus on the physical aspects of thecancer. Only some countries appear to address emotional or psychosocial needs effectively;see the Netherlands example on page 70.

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Period of Diagnosis

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Recommendations: towards better myeloma care in Europe

For doctors, nurses and the wider healthcare team:

• Ensure patients have access to the most up-to-date treatments in order to controltheir disease and offer the longest possible period of control, with optimal pain reliefto enable a good quality of life

• Facilitate quick, individual treatment decisions that are tailored to the individual,taking the patient’s views into account

• Ensure each patient has access to a multidisciplinary care team includingorthopaedic and renal specialists as well as a haematologist

• Generate stronger links between primary care and specialist centres• Ensure all suitable patients are made aware of all current clinical trials and given the

opportunity to participate if they wish• Take care to ensure effective communication with patients and their families, and to

give the patient sufficient time for dialogue• Psychological and psychosocial support and counselling should be available to help

all myeloma patients to deal with the emotional impact and with the stresses of dailyliving

• Provide access to as much, or as little information as patients want. This is especiallyimportant at diagnosis. Also provide tailored information and emotional support forcarers

For healthcare policymakers:

• Develop and implement national myeloma plans and awareness campaigns • Establish uniform care standards and a faster drug authorisation process• Support improvements to education for healthcare professionals, including GPs, on

diagnosis, symptoms and new treatment options for myeloma • Give priority to the elimination of differences between urban and rural treatment

options

Summary

It is clear that in many ways, myeloma patients in Europe suffer because of the rarity of theircancer. Health systems and health budgets are inadequate to meet their needs for access toup-to-date treatment and to all the medical disciplines that could help them, particularly inEastern Europe. There is also a great need for education of healthcare professionals ineffective communication and awareness of the urgent need of patients for information, theopportunity to play a real part in their treatment decisions and to participate in appropriateclinical trials.

3. Bw


3. Building a successful partnership with healthcare professionals



What do patients want from their doctors?

Patients feel positive towards their healthcare professionals when they appear to have aninterest in their care or “ask the right things”29. They can also feel intimidated by the medicallanguage, and are afraid of appearing ‘stupid’. From the doctor’s viewpoint, the main objectiveis usually to maximise the duration and quality of life for their patients. However, as well aspursuing those practical medical aims, the doctor must also be professional, honest, a goodcommunicator and provide emotional support.

Engaging in general conversation, sharing information and acknowledging patients’ knowledgein order to foster a more equal relationship can all help to build trust between doctors andpatients35. Setting out realistic, short-term and frequently reviewed goals, e.g. evaluating,prioritising and deciding on which actions to take, is a vital part of coping and helps patients togather the strength to face the uncertain future36.

Patients, on the other hand, are often as interested or more so in the quality of their life, ratherthan how well the treatment is progressing as shown by the level of biochemical markers.Some aspects of quality of life can actually be better than it was before diagnosis, as thediagnosis can drive people to address what is most important to them.

3. Building a successful partnership with healthcare professionals

Key points

• The ideal is a trusting, open partnership, with emotional support and opencommunication between patient and healthcare professionals

• GPs and specialists should be open to a joint decision-making process and empowertheir patients to share control of their treatment if they wish

• Treatment decisions should be made jointly and take account of each patient’spriorities and personality

• Setting realistic short-term goals and frequent review helps the patient’s ability tocope

• Treatment decisions should be reached only when the patient has had time to digestand absorb all relevant information and options

• Practical support for patients needs to be offered, and significantly strengthened inmany countries

• Information given orally about diagnosis, treatment and side-effects should be timely,well communicated and supportive; and supplemented by online or writteninformation

The patients and experts interviewed in the preparation of this report were of one voice instressing the massive importance of good communication between myeloma patients and theirdoctors and other healthcare professionals. Poor or ineffective communication has a majorimpact on how well patients are able to cope. This chapter explores what effectivecommunication can achieve, what stands in the way of good two-way partnership, and how therelationship can be nurtured to the benefit of both partners and principally that of the patient.



Again, the treatment decisions should take account of each patient’s priorities and character.While it will suit one person to seek less intensive or palliative care, others will call for “everytreatment you’ve got”. In addition, the diagnosis may end a long period of worry about what iscausing the patient’s health problems, enabling a more definite approach, and treatment mayrelieve pain – both of which can improve overall quality of life.

The impact of myeloma treatment on the practical aspects of life may also contribute tofeelings of anxiety and may raise additional questions and issues that need support beyondthe scope of healthcare professionals.

Patients will need to consider: will I be able to continue working/keep my income/keep mypension/ get travel insurance, and how will my family be affected? Practical problems likethese contribute significantly to the emotional burden of myeloma. Support from patientorganisations and social welfare bodies is essential to help patients deal with these issues andin many countries needs to be strengthened significantly.

In a more sensitive area, couples affected by myeloma, but considering having children, willneed extra information and support and guidance regarding the risks to fertility with sometreatments, as well as pregnancy prevention strategies during treatment with thalidomide orRevlimid® (see page 61). In addition, and very importantly, loss of a family member tomyeloma, especially a child losing a parent, will require support and possibly counselling forthe affected family members.

What issues get in the way of effective doctor-patient communication?

Shortage of time:One of the barriers to successful communication with hospital specialists is lack of time,although this need not be a problem if both specialist and patient are able to use the timeeffectively.

Trust:It is vital that patients trust their doctors29 and that the time and expertise are available andused effectively to give the right level of information and support. Providing the rightinformation, accounting for the benefits and the risks of the treatment, supports informeddecision-making, better adherence, less distress and a greater sense of control; plussatisfaction with the care received37. Feeling overloaded with information can lead instead toconfusion and worry29.

Communication skills are rarely taught in traditional medical training:Doctors’ ability to communicate with patients varies considerably. Ideally, training oncommunication skills should be added to the curricula of undergraduate medical schools. In recent years, continuing medical education approaches e.g. interactive online modules arebecoming available to enable healthcare professionals to improve their skills38.

Shared decisions

“…none of the doctors have undergone chemotherapy or a transplant themselves. That is why a patient is a very useful source of advice for the doctor, in the same way asthe doctor is an irreplaceable consultant for the patient in the treatment of multiplemyeloma …”

Slovak Myeloma Society Patients’ Club



For some other chronic conditions, a range of tools is available to facilitate communication andunderstanding between doctor and patient. These include use of wellbeing indexes e.g. WHO-5, to help define patients’ level of contentment or anxiety39.

Patients can be unsure how to communicate:Faced not only with a major shock at diagnosis, but also uncertainty over the future as a wholeand the individual ordeals they may have to endure, it is no surprise that patients often feeloverwhelmed by their situation. They need to absorb a lot of information; much of it quitecomplex and full of unfamiliar words, at a time when they are least able to formulatemeaningful questions and suggestions. This uncertainty is at the root of the common advicefrom people who have had myeloma for some time: to gather information and to become aswell-informed as possible, but it is not an easy path to take.

A full partnership

Professional, honest communication, a holistic needs assessment, and setting out realistic,short-term and frequently reviewed goals are crucial in discussions between patient anddoctor. This relationship enables a trusting partnership and equal participation in treatmentand management decisions – a vital way for the patient to cope and gather strength whenfacing an uncertain future.

Treating the person, not the myeloma

Trust between patient and doctor is essential because of the many decisions to be made,ideally in partnership. This is not only about deciding on the treatments being prescribed; thedoctor should ideally consider the whole person and how he or she is coping with theemotional stress of having myeloma.

In the UK, the National Institute for Health and Clinical Excellence (NICE) recommends that allcancer patients should have a holistic needs assessment at diagnosis, after initial treatmentand during follow-up40. A quantitative tool such as SPARC should be used, which enables thepatient to rate their pain, psychosocial, spiritual, financial and other issues, using a 4-pointverbal or Yes/No rating scale41; or the ‘distress thermometer’ which rates overall distress on anumerical scale42. Regular holistic assessment is an ideal that is met to a varying extent in thedifferent countries of Europe.

Attention to the patient’s preferences and state of mind goes a long way to improvingcommunication. This better sense of trust and understanding will also have practical benefits,for example patients’ compliance will improve if they feel that the doctor understands and issympathetic. Key situations where extra understanding is significant are:

Timing of diagnosis:Before receiving the diagnosis, the patient may have endured months with vague symptoms offatigue and bone pain; possibly compounded by feelings of being a time-waster. Oncediagnosed, they may also feel cheated by the time wasted and by not having had treatmentduring that time.



“Patients in our patient group report thatdoctors are very good at the medical aspects,but sometimes neglect the emotional side. Amyeloma patient is never well again – themyeloma will be there for the rest of his/her life.That asks for a very specific psychologicalapproach.”Greetje Goossens, representative of the Belgian patient groups and board member of MPE

Diagnosis:This is an extremely traumatic experience for most and needs time, care and attention to theneeds of the individual and acknowledgement of the seriousness of the news. At the firstmeeting, the patient is unlikely to be able to take in much detail, so the messages should bekept simple and supportive. It is important to stress that while myeloma is incurable, it iseminently treatable, that the prospects are improving rapidly and there is some hope for longerterm survival, particularly for younger patients. Spoken input must always be supplemented byproviding written or online information, at different levels of detail.

Treatment choices must be right for the individual:In discussing and deciding on the most appropriate treatment, patients need enoughinformation about the options and their benefits and side-effects. Importantly, they also needenough time to digest it before reaching a shared decision with their doctor. They need a gooddiscussion about the treatment options, the aims of each option and likely side-effects.

Shared decision-making:If the doctor is not open to the idea, it can be overwhelmingly difficult for even a patient who iswell-informed about myeloma to try to establish a debate about potential treatments. However,in general, patients need to become more empowered; developing the confidence andknowledge to seek a second opinion or to ask to be referred to a specialist treatment centre.Some patients report they had to “battle their way through the system”; having to seek severalmedical opinions before being offered certain treatments. Patients should also be able tochoose between treatment options, if they want to. Their views may also change as theirmyeloma progresses, e.g. if they become more ill or if personal circumstances change.

Not everyone wants all the details:Not all patients want to be actively involved in treatment choices, preferring to leave the detailsto the doctor. This view is particularly found in older patients and those in particular countries(as in Eastern Europe) or populations (e.g. Afro-Caribbean or Asian groups) where theaccustomed culture is that the doctor makes the decisions.

Summary

Effective doctor-patient communication is particularly important in order that patients retain asmuch control of their well-being as possible. Myeloma brings many challenges and if bothpatient and doctor are able to share both information and understanding that will contributegreatly to patients’ peace of mind and their quality of life.



Recommendations: Partnership with healthcare professionals

For patient organisations:

• Work to define patient expectations and needs when communicating with theirdoctors and other healthcare professionals

• Encourage individual patients to develop the knowledge and confidence to work inpartnership with their doctors in deciding on treatment; also to be able to challengerecommendations and to seek second opinions as a standard option

• Recognise that it will take time to change attitudes in both patients and doctors• Work with social security agencies, public awareness campaigns and the media

For doctors’ associations and teaching hospitals:

• Focus more on holistic care for myeloma patients, with as much attention toemotional wellbeing as physical therapy

• Urgently raise awareness of myeloma in young doctors entering primary care, inorder to reduce unacceptable delays in diagnosis

• Promote uptake of accreditation through the European Hematology Association forcentres offering training in blood cancer treatment, and enable implementation of up-to-date treatments and policies

• Devote greater attention in doctors’ training/continuing medical education to buildingcommunication skills, especially in conveying diagnosis, the relative risks andbenefits of treatments, and providing the appropriate level of information for theindividual patient

For healthcare policymakers:

• Support improvements in doctors’ training to include better communication skills andattention to the emotional as well as physical needs of patients

• Give priority to more satisfactory provision of emotional and psychosocial support toboth myeloma patients and their carers



4. Patients’ needs should be at the centre of research



Although a great deal is now known about myeloma and its effects, further research isessential to make progress towards establishing its causes, alleviating its symptoms, limitingits progress and ultimately finding a cure. As a rare cancer, myeloma research has sufferedfrom limited resources because of the relatively small numbers of people affected compared tothe major chronic conditions and other forms of common cancer. Due to the fact that myelomahas such devastating impact, patients are keenly interested to support research and to haveinput into research priorities.

This chapter examines the challenges and constraints facing research in myeloma andwhat patients’ priorities are for research, and the role they and their representativeorganisations can play to influence the agenda.

Take into account the needs of myeloma patients

…Put me, and others, back in a durable remission where we are symptom-free and treatment-free. How about eliminating peripheral neuropathy side-effects fromtreatment drugs? Get rid of all that myeloma bone pain, the broken bones, the backproblems. These things just debilitate so many myeloma patients over time.

Oh, and just cure the disease; that will take care of everything. I’m not holding my breathfor that.

Lou Ganim, Myeloma patient, USA*

* first published in Myeloma Beacon, 17 July 201243

4. Patients’ needs should be at the centre of research

Key points

• No current cure for myeloma means continued research is needed to find its causes,new effective and safer treatments and ultimately a cure

• High quality trials are needed to ensure statistical validity and best use of scarceresources

• Many patients are keen to take part in clinical trials and research• All patients should be made aware of clinical trials and given the option to take part if

feasible• Policy-makers should be continuously monitoring and benchmarking research

to achieve best possible outcomes• Patient organisations should be involved to identify research priorities and objectives

to help ensure that limited resources are directed towards patients’ priority areas• Patient groups and individual patients should advocate better collaboration and

coordination of research projects, to reduce duplication, make best use of availableresources and deliver research outcomes

• Interpretation and application of legislation needs to be simplified and standardisedacross Europe – the current proposal for an EU regulation on clinical trials gives theopportunity to ensure simpler procedures and more transparency, which shouldreduce the regulatory burden on trials and encourage more to be conducted inEurope

• Patients’ views on clinical trials should also be reported and taken into account



Why is research so critical for myeloma patients?

Once diagnosed with myeloma – even with smouldering myeloma which has no symptoms –patients know that there is no cure. As myeloma is a relapsing and remitting cancer, there is apressing need to understand the mechanisms that trigger its stop-start stages of progression.

Treatment can do a great deal to relieve the symptoms and temporarily halt the progress ofmyeloma and research in the last ten years has resulted in the development of lenalidomide(Revlimid®) and bortezomib (Velcade®) that can improve the performance of chemotherapydrugs when given in combination. More new treatments are keenly anticipated.

In addition, some of the traditional treatments themselves have unpleasant side-effects. Forthese reasons, continued research is urgently needed, as myeloma patients are all working ona limited timeframe. That is why they are especially keen that the rapid advances of researchof recent years are translated into treatments.

What types of research are being undertaken?

Research is continuing on potential new treatments and to assess interventions to reducetoxic side-effects from existing drug regimens. Other types of research focus on the impact ofmyeloma on the quality of life of patients; or assess the true and economic value of treatmentto patients, health systems and society as a whole. This is often done through a process calledHealth Technology Appraisal.

Genetics is a vital area of current myeloma research; focusing on defining the factorsresponsible for the onset and progression of myeloma, identifying its sub-groups and thefactors responsible for both relapse and refractory myeloma (myeloma that no longerresponds to a specific treatment). It is also likely that a person’s genetic makeup influencestheir susceptibility to environmental risk and their response, but much more work is needed inthis area44.

There is no single genetic change that pinpoints the tendency to develop myeloma45, but it isbecoming possible to stratify treatment more closely to individual patients46. Here, researchand development of genetic techniques is as important as development of new drugs.

Among recent advances in these techniques are cytogenetic analyses of myeloma cellchromosomes, which can detect any sections that have become broken and rejoined to otherchromosomes (translocations), or where myeloma cells have an abnormal number ofchromosomes or other abnormalities47. Fluorescence in situ hybridisation (FISH) studies ofbone marrow plasma cells can detect translocations and other chromosome changes toosmall to be detected by other cytogenetic testing47, identifying patients with high-risk myeloma.Gene-expression profiling (GEP) is another technique capable of classifying patients as high-risk or low-risk45,46. All of these technologies enable a more accurate prognosis to bedeveloped and appropriate care selected.

Those at high risk (with expected survival of less than three years) could justify more intensivetreatments like allogeneic stem-cell transplant or trials of innovative experimental drugcombinations46. Those at low risk (likely to survive for 7-10 years or more) could more suitablybe offered less aggressive treatments that focus on minimising toxic side-effects andoptimising quality of life46. However, the decision is not clear-cut and the approach should bedecided on the basis of the individual patient’s needs. In any case, these tests are difficult tointerpret and need rigorous quality control which is possibly not yet achievable on a widescale45. It is likely that in the future, GEP techniques can be developed so that it will becomepossible to identify specific treatments that will get the best response from a given patient45.



Clinical research, e.g. on the mechanism of action of a potential new drug or to assess theaction of drug combinations, follows the following stages:

Pre-clinical laboratory research:This uses myeloma cells in culture and animal models to establish if a drug is likely to work,without causing major side-effects. If laboratory results indicate that a drug candidate ispromising and safe, it will undergo clinical trials. These can also investigate the use of newcombinations of drugs or new treatment regimens.

Clinical trials/development:Clinical trials are conducted to test the safety, efficacy of treatments or combinations oftreatments; first in small pilot studies and later in trials involving many hundreds of people oreven more. They are carried out according to strict procedures (protocols) which have beenapproved by an independent research ethics committee to protect the interests of theparticipants. Currently a wide range of trials is under way in Europe48, investigating the role of:

• current myeloma treatments e.g. bortezomib (Velcade®), thalidomide, lenalidomide(Revlimid®)

• newer proteasome inhibitors e.g. carfilzomib and immuno-modulatory drugs e.g. pomalidomide

• novel targeted therapies e.g. antibodies, signal transduction inhibitors, targetedradiotherapy and vaccine approaches

• supportive care issues: bone complications, infections, painClinical trials are usually carried out in hospitals and may be funded by a public sectorresearch body (e.g. the EU’s Research Framework Programmes, the UK Medical ResearchCouncil), by a private foundation (e.g. the International Myeloma Foundation) or research-funding organisation (e.g. Myeloma UK, the Multiple Myeloma Research Foundation or theEuropean Myeloma Network) or by a pharmaceutical company.

Since the mid-1990s, the EU has contributed funding to some 25 collaborative researchprojects on aspects of myeloma under the Seventh and earlier Research FrameworkProgrammes. Current EU-funded projects relevant to myeloma are examining the molecularcauses of drug resistance (led by a research team in Belgium), optimising therapeutics in high-risk myeloma (Austria), and cancer-associated bone disease (UK)49.

Real-world dataA further aspect of research is the study of real-world data and the analysis of myelomaregistries, which collect data on the epidemiology of myeloma in the population as a whole.Analysing registries can be the source of wider understanding of how medical conditions affectlarger numbers of people – especially important for a rare disease like myeloma. They giveresearchers the opportunity to examine possible relationships between the incidence andprevalence of diseases, and environmental or population factors.

What are the challenges and constraints facing research in myeloma?

Funding and prioritisation:It is vital that research is directed towards efforts that will make the greatest improvement tothe lives of myeloma patients. Pharmaceutical industry researchers in particular, who accountfor 79% of the trials done each year in the EU50, should focus on developing research projectsof the highest quality; large enough to ensure statistical validity, and with real potential to makea difference to patients’ lives.



Patient organisations can help by identifying priorities and objectives and by making the casefor adequate, prioritised funding for research studies in both the private and public sectors.The objective of achieving the best possible outcome from investment in research should becontinuously monitored and benchmarked by health policymakers.

Legislative control:Clinical trials in the EU are regulated by the requirements of the EU clinical trials directive52

which is designed to ensure that no harm comes to the participants and that the outcomes arescientifically validated. However, a recent review of the directive has shown that itsrequirements have been interpreted and applied differently in different member states; theyhave doubled the cost of running trials since before it came into force, and the number of trialsbeing conducted fell by 25% between 2007 and 201153.

“The market economy fails to provide adequately for research in all cancers. This is a keyarea where Europe can add value by supporting academic and translational research. We can use funds from the Seventh Framework Programme to encourage research andinnovation in the areas of primary prevention, screening and early detection, and new anti-cancer medicines and treatments, particularly in the realm of rare and difficult-to-treatcancers which may be considered unprofitable by industry… we can revise the politicalframework to stimulate further research.”

Adamos Adamou MEP,

Co-Chair of MEPs Against Cancer*, 10 April 2008

* MEPs Against Cancer

These difficulties led to a new measure being proposed in July 201254 – this time a regulation.As a regulation, this will have direct legal force in the member states, unlike the previousdirective which had to be implemented by national legislation; often leading to differentinterpretations in the different countries.

The proposed regulation will offer simpler procedures and more transparency about recruiting;it should come into force in 2016. The European Commission intends that this change shouldreduce the procedural burden on trials investigators having to meet varying requirements inEurope, and encourage more trials to be conducted in Europe.

Reducing duplication:Research into cancer (of all types) and its treatment is diverse and fragmented across the European Union. The European Commission has called for collaboration and better co-ordination of research in order to cut down on duplication and enable faster progress55. Itsown Seventh Research Framework Programme is funding several collaborative clinical trials56.

Translating research results into clinical practice:Patient organisations can support the best implementation of research by highlighting togovernment agencies the most promising outcomes from clinical trials, so that researchfindings can rapidly be adopted into clinical practice. While this should not be the responsibilityof patient organisations, initiatives from them can only be a step in the right direction.

Rare disease research:As one of the rarest forms of cancer, development of myeloma treatments has suffered fromthe relatively small market for its treatments. Low patient numbers challenge trial organisers to assemble statistically valid results. However, patient advocacy as a whole must continue todraw attention to the urgent therapeutic needs of those with a rare disease.



‡‡ IMI is a joint undertaking between the European Commission and the European pharmaceutical industry association EFPIA, that sets a strategic research agenda for innovative medicines, with joint funding

The Innovative Medicine Initiative (IMI)‡‡ has established that it would be useful to support anIMI topic on co-ordinating R&D and enrolling studies on rare and neglected diseases, but thisis not yet set up57. IMI aims to focus on defining strategic themes with maximum impact and‘game-changing’ ideas and areas where the maximum number of companies can join forces inco-ordinated research58.

Patients’ views on trials:Rigorous control of the conduct of trials has made it hard for patients’ views to be heard,despite ethics committees including a member who is a patient or representative of a patientorganisation. Patient input can clearly be significantly increased – both on the review of thelegislation itself and on ensuring the relevance to patients of the measured outcomes ofindividual trials.

Currently two EU-funded FP7 collaborative projects are of particular relevance:56

• PatientPartner (Identifying the needs for patients partnering in clinical research) whichaims to involve patients’ organisations so that research can be better adjusted to thereal needs of patients, and

• PREDICT (Increasing the participation of the elderly in clinical trials) investigates whythe elderly are under-represented in clinical trials and what can be done to improve theirparticipation.

Let’s hear the bad news too:In industry-sponsored research, negative trials outcomes have historically not always beenpublished but this is now changing. Patient organisations and other researchers need to knowabout negative findings as well as positive, in order to acknowledge treatments that are noteffective or have worse side-effects and to save wasteful duplication of non-productiveinvestigations. The proposed clinical trials regulation will require results to be reported to thenational authorities within a year of the trial’s completion; a feature that deserves solid supportfrom patient organisations.

Furthermore, the website http://www.clinicaltrialsregister.eu will soon publish all results(positive and negative). This is likely to happen in the second part of 2013.

Use of biological resources:It is important that as well as using financial resources to the best possible outcome, patientswho have contributed tissues for laboratory studies should be confident that their tissue isbeing employed in top-end research that should ultimately benefit patients.

Patients’ priorities

Patients do not always have the same view of what is needed in myeloma research as thatheld by researchers. Priorities for patients are that research should be patient-centred: ideallyit should provide strategically relevant evidence from which patients can benefit directly.

Patients are very keen to take part in clinical trialsMany patients are interested in participating in clinical trials, as the treatments being evaluatedmay well help to control their myeloma better or improve their quality of life.

They must be referred by a doctor, but many online websites give details of trials that arecurrently recruiting patients. Among these are the EU Clinical Trials Register, which givesaccess to the EudraCT database used by national medicine regulatory authorities to enterclinical trial data; and the US database§§.



Ideal priorities for clinical trials – a patient perspective

• Identify practical alternatives to corticosteroids• Identify optimal treatment for elderly, high-risk patients• Find a solution for peripheral neuropathy – a side-effect affecting up to 75% of

patients treated with bortezomib and thalidomide• Allow “patient-friendly” studies (regardless of whether they are industry- or

investigator-initiated) to become the basis for drug authorisations, as these takepatients’ needs into account

• Publish up-to-date information on trials• Conduct psychosocial research alongside clinical trials

The outcomes of research should inform clinical and practical decisions whether to offerpatients new treatments, or treatments modified in some way that improves the prospect ofremission or reduces one or more side-effects. It should be ethical, non-competitive, andprioritised.

Contact between patient organisations and trials groups is essential to make sure thatresearchers share these priorities and many clinicians would welcome this input, as patientsare the recipients of treatment and the bearers of side-effects.

Some of the key priorities for patient organisations in relation to seeking more patient-centredresearch are:

Identifying a practical alternative to corticosteroids:This relates particularly to dexamethasone; currently a major treatment component but withunpleasant side-effects including weight loss and muscle wasting or weight gain, bloating,irritability, personality changes, hyperactivity and insomnia.

Identifying optimal treatments for elderly, high-risk patients:Older patients are typically less fit and less able to tolerate intensive chemotherapy treatmentfollowed by stem cell transplantation. They are usually offered less invasive (and lesssuccessful) treatment. This group of patients is therefore in great need of more satisfactoryalternative treatments.

Finding a solution to peripheral neuropathy:A common, distressing symptom of myeloma is pain, tingling, and numbness in the hands andfeet as a result of peripheral neuropathy. It can be apparent at myeloma diagnosis, but affectsup to 75% of myeloma patients treated with bortezomib and thalidomide59.

Flexibility for off-label drug use in clinical trials:Patient organisations are best placed to investigate with the European Medicines Agency(EMA) the possibility of more flexibility for clinical trials requiring a combination of new drugsthat will be compared with existing authorised products (“reference drug or comparator”).Normally, authorised medicines have to be used within their licensed authorisation for dosingand schedule.

Since its marketing authorisation, small changes in the dose or schedule of the reference drughave often been discovered that allow less side-effects without reduced efficacy.

§§ http://www.clinicaltrialsregister.eu/ and http://www.clinicaltrials.gov/



In a clinical trial, experimental regimes can be tested, also for the licensed drug but the studyproposal needs to clearly explain the rationale for a different dose and/or schedule and what itmeans in terms of safety and efficacy for the patients participating in the clinical trial. At thisstage of clinical trial development, interaction with patient organisations – who can bring thereal-word patient needs in the discussion – might be very valuable.

Publication of up-to-date information about trials:It is of utmost importance to patients that plans for trials and their outcomes are published.Information on trials in the 27*** EU member states is already published in the EU ClinicalTrials register†††. As not every country in Europe is conducting relevant trials, publication ofresults, as will be required by the coming EU regulation, will ensure that myeloma patients andtheir healthcare team can be fully informed about current trials and their scientific and clinicalfindings. The European Medicines Agency is currently examining and consulting on thepractical and policy issues involved before the data it holds can be made available in the mosthelpful form to serve both patients and public health authorities60.

Research that can lead to personalised care:This is a vital aspect, because patients vary substantially in their response to differenttreatments and even in their response to a single medication being given at different stages intheir progress. For this reason, the genetic causes of myeloma are being investigated closely;notably by the Multiple Myeloma Genomics Initiative of the Multiple Myeloma ResearchFoundation that has led to the complete mapping of the myeloma genome61 and theCoMMpassSM study that will follow 1,000 newly diagnosed patients to determine the molecularand genetic changes underpinning the evolution of the disease62.

Psychosocial research:Alongside clinical trials are qualitative studies of patients’ and carers’ emotional andpsychological needs, and strategies to support them. Patient organisations are calling urgentlyfor more activity in this area, as they have access to the personal experiences of thoseaffected by myeloma. Here also is the opportunity to argue for dedicated training and raisedawareness among healthcare professionals.

Inviting myeloma patient groups to the table

Patients and patient organisations can play a huge and multi-faceted role in both laboratoryand clinical research. Evidence suggests that this resource is being under-utilised, possibly tothe detriment of other stakeholders in research. Patients and patient organisations caninfluence and assist the research agenda in many ways, including:

Promoting research and raising awareness:Access to trials appropriate for the individual will clearly vary in different countries, accordingto the availability of suitable centres and research clinicians. Here there is a clear liaison rolefor patient organisations, which are ideally placed to provide the patient’s perspective to theresearchers, and to inform both patients and researchers about the practical needs for andavailability of trials.

Access can depend on the patient’s level of initiative in searching internet databases, or theinvolvement or lack of involvement of the specialist in trials, or simply the availability of a trialappropriate to the patient at the right time in a reasonably accessible place.

*** 28 Member States from July 2013, when Croatia will become part of the EU††† http://www.clinicaltrialsregister.eu/



Advocating policy changes:Patients and their representatives have a valuable contribution to make to the mechanismsthat authorise clinical research – the ethical committees and the protocol developers and toproduct market authorisation.

At the European level, patient representatives participate and have full voting rights in thework of the European Medicines Agency (EMA) and a number of its committees relevant tomyeloma including the Committee for Orphan Medicinal Products, of which the vice-chair is apatient representative63, the Committee for Advanced Therapies and the PharmacovigilanceRisk Assessment Committee.

Patient representatives also contribute to EMA advisory groups in the context of promotingdrug development, ensuring clear information to patients and working towards equal access totreatments at European and national levels.

Rare disease patients are invited to contribute as experts on the development of productsappropriate to their disease, e.g. in protocol assistance (development). However, thisinvolvement of patient representatives within EMA does not touch on pricing andreimbursement, which are not within the EMA remit.

At a broader level in the EU, the EMA has established its Patients’ and Consumers’ WorkingParty with members from a wide range of European patient organisations. A major example ofthe contribution of patient organisations in obtaining product authorisation is that ofthalidomide and Revlimid® (lenalidomide) for use in myeloma in Europe; however even afterEMA authorisation, the conditions for access in individual countries vary significantly64.

At national level, permanent patient representation on national regulatory committees is veryvariable between the countries and non-existent in many. Here, as well as supporting thesame European objectives, patients’ representatives would also address pricing andreimbursement for treatments.

Adoption and implementation of research outcomes:A further area of invaluable contribution from patients is that of health technology assessments(HTAs), which are used in many European countries by healthcare authorities to reviewevidence and guide decisions on the value for money or cost-effectiveness of varioustreatments.

HTA bodies review the evidence of scientific studies, so it is very important for this to besupplemented by information from patients and carers on their real experience of thetreatments in practice. This impact can include not only whether symptoms were alleviated,but to what extent, at what cost in terms of side-effects and what benefit the treatmentenabled, that was not possible without it65.

Summary

Research is a crucial area of interest to myeloma patients; potentially promising great rewardsfor the future but fraught with regulation, high cost and lack of focus on the patient. Thecoming EU regulation on clinical trials promises to reduce the administrative burden onresearchers, so hopefully will inspire more clinical trials to be conducted in Europe and enablemore European patients to benefit. Its effects will, however, take years to be felt as it onlycomes into force in 2016. In the meantime, patient organisations have an important role toplay in making known patients’ priorities, and their urgency, to both policymakers and thoseconducting and funding research in both the public sector and the pharmaceutical industry.



Recommendations: Research

For all stakeholders:

• Aim for greater collaboration between the different stakeholders involved in researchin order to develop strategies that meet all their varying objectives and to reduceduplication

• Support and invest in collaborative research e.g. through joint projects, EU-funded collaborations between industry and academia and undertakings such asthe Innovative Medicines Initiative

• Direct research towards initiatives that will make the greatest improvement to thelives of people living with myeloma


• Highlight to government agencies the most promising outcomes from clinical trials,so that their findings on new therapies can rapidly be built into treatment guidelines

• Give positive support to the aims of the proposed EU regulation on clinical trialstowards creating more equal access for patients to clinical trials throughout Europeand to encourage trials investigators to conduct their research in Europe onceprocedures have been made more uniform

• Take the opportunity presented by the new clinical trials regulation to call for morepatient representation in the control of clinical trials and to emphasise that new drugsshould truly address the needs of myeloma patients and not just the requirements formarket authorisation

• Advocate urgently for more research on patients’ needs for psychosocial support andfor dedicated training and raised awareness of healthcare professionals of theemotional needs of patients and carers

For market authorisation authorities (European and national):

• Monitor and benchmark the quality of research studies in order to ensure thatpatients' needs are reflected in the outcome of investment

For international research foundations:

• Continue, and where possible extend, their support for clinical research intoalleviating and ultimately curing myeloma

• Continue and extend their interaction with patients in order to highlight the greatestpriorities for future research funding

For the pharmaceutical industry:

• Increase mechanisms for interaction with patient representatives in the concept anddesign of clinical trials

• Prioritise studies focused on patient needs • Publish negative findings as well as positive, in order to acknowledge treatments that

are not effective or have worse side-effects than existing treatments



5. An effective policy framework for treatment and care in myeloma care



Although not responsible for health policies or for providing healthcare in the member states,the European Union does play an important role in encouraging measures to improve publichealth and to co-ordinate national efforts. EU influence is important because its measures topromote centres of expertise and European reference networks will have particular benefit forrare diseases such as myeloma, where patient numbers are so few that there is a need forconcentration of cases in order to ensure that the units or centres dealing with myeloma havethe appropriated expertise. A highly co-ordinated system of referral and coordination of carewould also be needed in order to guarantee the accessibility of patients who live in more ruralor remote areas, since they can experience difficulty in reaching expert medical attention. Alsohigh on the EU public health agenda are the aims to stimulate national cancer plans and to co-ordinate registers and databases.

5. An effective policy framework for treatment and care of myeloma patients

Key points

• Rare diseases, including myeloma, have been a priority for action in the EuropeanCommission’s health strategy for 2008-2013; reinforced by measures to improveaccess to orphan drugs and incentives to promote research into rare diseases

• However, not all member states ensure full access to every orphan drug, regardlessof authorisation by the European Medicines Agency

• Key aspects of the EU and national policies relating to myeloma:o Identify and foster Centres of Expertise and improve the diagnosis and care

capacity in member stateso Create European reference networks and facilitate the participation of

National Centres of Expertise to ensure patients’ access to medical expertise, information and to improve diagnosis, care, training and research

o Develop uniform national cancer planso Create guidelines for myeloma careo Give timely and fair access to orphan drugso Ensure transparent and regulated drug pricingo Employ health technology assessmentso Maintain comprehensive registers and databases

• Patient groups need to act and make input to clinical guidelines to ensure the EU legislation is effectively implemented locally, in order to;o Secure fair access to medicines for patients,o Avoid duplication of processeso Give timely access to orphan drugso Improve diagnosis and care management



The current policy landscape in Europe

In Europe, national governments are responsible for defining their health policy and fororganising and delivering health services and medical care. The European Union does,however, have the role of complementing national policies and encouraging co-operationbetween the member states in improving public health, preventing physical and mental illnessand diseases and obviating sources of danger to physical and mental health66. As such, itcontributes both legislation and non-binding recommendations which are directly relevant tomyeloma care.

The EU has been active on rare diseases, including myeloma, since 1999, and they wereidentified as a priority for action in the European Commission’s health strategy for 2008-201367. Current EU policy, set out in the Commission’s communication of 2008 and the Councilrecommendation of 2009, supports national policies including national action plans, andencourages co-operation between member states and better information exchange68.

Crucially, the action plan calls on member states to support centres of expertise (also calledcentres of reference or excellence at member state level) and European reference networks inorder to improve universal access to high-quality care for rare diseases68. The criteria andconditions to be fulfilled by ERNs and CoE are now being developed by the EuropeanCommission, in compliance with article 12 of the cross-border healthcare directive.

This step is reinforced by measures to improve access to orphan drugs‡‡‡, and possibleincentives to promote research into rare diseases and development of orphan drugs68,69.However in practice, not all member states ensure full access to every orphan drug authorisedfor the market70.

The Council recommendation on rare diseases§§§, a political commitment of member states,called for member states to develop national plans for rare diseases by 2013 at the latest; tocontribute to information resources, to identify needs and priorities for research and supportnational participation, to support the creation of centres of expertise, to employ nationalexpertise to contribute to European guidelines, training and sharing of best practices, and toencourage empowerment of patient organisations71.

The European Union Committee of Experts on Rare Diseases (EUCERD****) was set up in2009 to support European Community actions in this area. The European Commission alsofunded the European Project for Rare Diseases National Plans Development (EUROPLAN††††)between 2008-2011, and recently has funded in agreement with the member states a jointaction on rare diseases (2012-2015) which will take forward and complement the work carriedout by EUROPLAN, giving support to national health authorities in their development andimplementation of national plans for rare diseases.

‡‡‡ Orphan drugs are those intended for the diagnosis, prevention or treatment of a life-threatening or chronically debilitating condition affecting not more than 5 in 10,000 people, i.e. a rare disease

§§§ Council recommendations call for a particular course of action but unlike directives or regulations, have no legal obligation

**** http://www.eucerd.eu/__PP__1.html†††† http://www.europlanproject.eu/_newsite_986987/project.html



Key aspects of European and national policy

Centres of expertise:The idea of developing centres of expertise, raised in the EU’s 2008 communication, wasagain reinforced by the European Parliament’s 2010 resolution stressing that special action isto be taken for rare and less common cancers.

The Directive on patients’ rights in cross-border healthcare specifically addresses the topic ofEuropean Reference Networks and Centres of Expertise in its article 12, providing a first legalbasis for the implementation of networks and common criteria for the recognition of Centres ofExpertise at EU level. The aim of the centres and networks is to share expertise with othercentres across the EU and to establish networks of knowledge and care and herewithaccelerate diagnosis and make expertise more widely available72. These centres will complywith strict criteria and conditions (which are being developed by the European Commission atthe moment) and offer services specialised, i.e. for single or a group of rare diseases.

In some countries there are already similar systems of designation in place. However,although the criteria for designation are in line with the EUCERD recommendations73, they dovary between countries70.

National centres of expertise in Europe (December 2011)

Source: 2012 Report on the state of the art of rare disease activities in Europe, of the European Union Committeeof Experts on Rare Diseases70

CE designated in the context of a national RD plan/strategyCE designated outside of a national RD plan/strategyCE officially recognised to varying degrees but not designatedCE by reputation only/self declaredCountries with plans to designate centres with a national RD plan/strategy



European reference networks:In 2008, the European Parliament specifically called on the European Commission to usefinance from the structural funds and the Seventh Research Framework Programme to set upand fund reference networks for rare and difficult-to-treat cancers, in order to pool resourcesand expertise and improve diagnosis and treatment74. The Commission responded with itsEuropean Partnership for Action against Cancer for 2009-201355, to co-ordinate actions takenby the member states and other participants. This would be followed by a new joint action oncancer (2013-2016) which will complement the work already done.

The Commission’s 2009 recommendation called on the member states to encourage theformation of reference networks to link the centres of expertise in rare diseases, and toimprove access to diagnosis and quality healthcare. The directive on patients’ rights to cross-border healthcare75 sets the objective that European reference networks can improve theaccess to diagnosis and the provision of high-quality healthcare to all patients who haveconditions requiring a particular concentration of resources or expertise.

These networks were designed to encourage the exchange and co-operation amongprofessionals and between the centres of expertise. They are also focal points for medicaltraining and research, and for dissemination and evaluation of information, especially for rarediseases. The directive gives the Commission’s undertaking to develop the conditions andquality criteria for the reference networks75. From the patient’s viewpoint, the networks are avery valuable advance, as they enable them to benefit from the knowledge and expertise ofmedical researchers in other member states.

As yet there are no designated reference networks directly relating to myeloma, but pilotevaluation has been made of networks in 11 other disease areas, identifying issues to beaddressed for future networks76.

It is important that patient organisations are vigilant during the development, during 2012-14,of the mechanism to set up the European reference networks. This is a major opportunity forpatient organisations to influence their national governments and the European Commission totake into account patients’ needs, for example, in relation to research. The inclusion ofresearch professionals should be decided by strict but neutral criteria.

National cancer plans:National Cancer Plans, advocated in 2009 by the European Commission’s communication onaction against cancer55, are public health initiatives intended to reduce cancer incidence andmortality and to improve the quality of life of cancer patients. To do this they use evidence-based actions for prevention, detection, treatment, palliative care and research77. High amongtheir objectives are to promote the development of care management guidelines and toemphasise the importance of early diagnosis. Some member states have not even begunwork on national cancer plans and they are still under development in others. A 2009 study ofnational cancer plans in 31 European countries found only 19 functioning national plans (ofwhich four were for the constituent countries of the UK)78 and significant differences remainedbetween them. Gaps were evident in many, in respect of governance, targets, timelines andfinancing78.



Access to drugs for rare diseases:• Critical medicines are still not available. Drugs to treat rare diseases must be both

available and affordable. In the EU, there are many examples of critical medicines notbeing available, for example in Romania, while in Poland access still depends onnegotiation between the pharmaceutical industry and the reimbursement authority

• A call to establish common pricing and reimbursement decisions. A proposal wasdeveloped in 2009 by the European Organisation for Rare Diseases (EURORDIS), withindustry and academic leaders to call on the European Commission and the EuropeanMedicines Agency to establish a working party for European collaboration towardcommon scientific assessment of the clinical added value of orphan drugs79. Theintention was to establish a common basis for national pricing and reimbursementdecisionsThree surveys were conducted by EURORDIS in 2004-10, and the EU PharmaceuticalForum concluded that “equitable and timely access to approved drugs for rare diseasespatients remains an issue and effective market access and utilisation vary stronglybetween and within member states”80. The European Commission mandated a studyfrom Ernst & Young, which provided the basis for the 2012 recommendation of the EUCommittee of Experts on Rare Diseases80. The recommendation sets out a mechanismfor information exchange between member states in order to help them to makeinformed decisions on access to orphan medicinal products and, most notably, to bridgethe knowledge gap at the time of marketing authorisation81

• Is sharing the risk a viable answer? One alternative approach to financing orphandrugs is to share the risk. This has been used in the UK for providing bortezomib(Velcade®) to treat myeloma. The arrangement is that the National Health Service paysfor the drug only when patients show a full or partial response to treatment after fourcycles of treatment. If not, the manufacturer will refund the full cost or provide areplacement product. Such arrangements give access for the patient and minimise thecost to the healthcare system, but their effectiveness in improving access to newproducts has not yet been properly evaluated82

• Early approval for severe conditions with significant unmet need. The EMAapproval process is very relevant to the availability of drugs for myeloma, with increasedpressure for its mechanism of early approval in cases of severe conditions withsignificant unmet need83. In addition to the normal comprehensive procedure, the EMA isable to grant early, conditional approval in cases where it is possible to demonstrate apositive benefit/risk balance based on preliminary evidence. This type of approval,renewable annually, carries the absolute obligation to provide the missing data towardsa normal market authorisation84. This is an area where patient organisations are bestplaced to advocate for wider use of conditional approval, both in specific cases and as ageneral principle

Drugs pricing:• Objective, non-discriminatory pricing for cross-border healthcare. Pricing for

medication is negotiated between the national pricing and reimbursement authoritiesand the pharmaceutical industry. The cross-border healthcare directive requires thatdrug pricing information is given clearly and that the same fees as for domestic patientsare charged to those from other member states. If there is no comparable price fordomestic patients, the price should be calculated according to objective, non-discriminatory criteria



• A call for the cross-border healthcare directive to safeguard patients’ access to

medicines. A great deal depends on the effective transposition and implementation ofEU legislation within the member states, as drug pricing directives have to betransposed into national law. Access to medicines can be impeded for less affluentpatients in countries where the patient is required to pay up-front, and be reimbursedlater. Patient organisations have until October 2013 to call on their national governmentsto transpose the cross-border healthcare directive in a way that effectively safeguardsthe needs of patients

• Measures regulating the prices of medicinal products to be transparent. Also ofgreat significance is the amendment proposed in March 201285 to the 1989 directive ontransparency of measures regulating the prices of medicinal products. The originaldirective regulates the power of EU member states to set the pricing and reimbursementconditions for medicines, but had become seriously outdated. The proposed amendingdirective will replace the original, extend its pricing and reimbursement controls to coverhealth technology assessments (next paragraph) and reduce the time limit for pricingand reimbursement decisions to 60 days, which should improve patients’ access to newmedicines

Health technology assessment (HTA)• Methodologies for HTAs to be shared across borders. The cross-border healthcare

directive gives the European Commission’s support for a voluntary network connectingnational authorities or bodies responsible for health technology assessments designatedby the member states (see section 4: Research). The network should avoid duplicationand support collaboration between member states in developing and sharingmethodologies for health technology assessment, including relative effectivenessassessment75

Guidelines for myeloma treatment and care• A need for uniform clinical guidelines. These are intended to guide decisions in

diagnosis, management and treatment in specific therapeutic areas, based on the bestavailable evidence and consensus. Difficulties can arise through differing approaches ofguidelines from different sources. At present, standards vary because of the rarity ofmyeloma and consequent delay in diagnosis and because of differing pricing andreimbursement structures. European treatment guidelines and consensus statementshave been published and are regularly updated, but the presence of too many can dilutetheir value86,87

• Myeloma Patients Europe to contribute to the development and application of a

uniform European consensus on optimal care. This is a key aim of the group, so thatmyeloma patients can receive the same high level of care throughout Europe. The keylimitation to this aim is the different national reimbursement decisions which may restrictthe availability of some medications. The UK Myeloma Forum, which developed the keyguidelines on diagnosis and management of myeloma and supportive care, is keen towork with the National Institute for Health and Clinical Excellence (NICE) to developNICE guidelines on myeloma management. Such guidelines would be highly influentialin the prospects of developing European guidelines



Registers and databases• Data is essential, but epidemiological data on individual rare cancers like

myeloma are very few. The Orphanet database shows 28 myeloma registries in only15 EU member states88. Data on the epidemiology of myeloma is essential in order toexamine its incidence across Europe, to reveal the discrepancies and to identify regionsof better practice in order to share effective strategies89. Orphanet and other databasesalso provide invaluable information on current clinical trials (see page 50).

Policy solutions to lift the barriers to excellent myeloma care

Clearly a great number of initiatives are already in place both nationally and at the Europeanlevel, but patients are not receiving a uniformly high level of care. So what policy solutions cancontribute to breaking the barriers to achieving excellence in myeloma treatment and care?

• Access to novel medicines – While not questioning the sovereign right of nationalhealth authorities to manage their reimbursement policies, there is room for EU-levelrecommendation that variation in national reimbursement policies, which damages thetreatment prospects of patients, is removed. The onus is also on the pharmaceuticalindustry: not to place medicines beyond the reach of health authorities on the grounds ofprice

• Access to optimal care – Uniform guidelines for myeloma diagnosis, management,treatment and supportive care that were fully implemented across Europe would be amajor contribution to raising standards and improving equity. National cancer plans arerunning or under development in most member states but show significant shortcomingsin terms of governance. To be effective, they should not only set targets, but be followedby regular, independent assessment including evaluation of results and targets achievedor missed

• Centres of expertise and European reference networks are at an early stage ofdevelopment and need effective co-ordination, evaluation and extension into the otherareas including blood cancers; but they should eventually contribute to patients’ accessto the highest level of treatment. Where it is not possible for patients to reach suchcentres because there are none in their own country, the reference networks should befully employed to enable transfer of staff or at the least, electronic consultation andtransfer of expertise. Also, good communication between centre of expertise and otherlocal specialised and non-highly specialised healthcare providers (follow up, palliativecare, etc.) is essential for patients that are not receiving highly specialised care at thatmoment but need specific attention (weak immune system, etc.)

• Access to cross-border healthcare – Patients prefer to be treated in their country ofresidence, but when this is not possible or it is taking too long to get the diagnosis ortreatment, they have the right to cross-border healthcare (as provided by the 2004 EUregulation on the co-ordination of social security systems90 and the cross-border caredirective). However, even under these improved conditions of access, patients who go to other countries for treatment may still bear the responsibility for finding information onpotential treatments, demonstrating to insurers that the treatment has been carried outand submitting the correct documentation91.The European Commission estimates thatthe cost of total cross-border healthcare is about 1% of EU public healthcare spending92

– a small proportion but vital spending for rare diseases



ACCESS is the key for patients

Optimal care depends on patients’ access to:• Novel medicines• Effective care plans• Cross-border healthcare• Expertise• Uniform guidelines• Clinical trials• Early diagnosis• Opportunity to input patients’ priorities into policy making.

‡‡‡‡ http://ec.europa.eu/health/files/clinicaltrials/2012_07/proposal/2012_07_proposal_en.pdf

The directive on cross-border healthcare provides that national governments maychoose which reimbursement mechanism is to be used for care provided to patients from other member states, and include it in their legislation before 2013. Patientorganisations should call on their national governments during this time of preparation ofnational laws to implement the directive to select a direct payment from the memberstate of residence of the patient, to the member state of treatment. The alternative is forthe patient to have to pay upfront, and then reclaim the amount from their own memberstate.

• Access to trials is not currently available in every European country, because of therarity of myeloma and the availability of research hospitals. The recent proposal for aRegulation on clinical trials on medicinal products for human use‡‡‡‡ (which, if adopted,will repeal the currently applicable Directive 2001/20/EC on clinical trials) would worktowards further harmonisation in the area of clinical trials, facilitating the process ofcarrying out clinical trials in several EU member states at the same time and thereforeimproving patients’ ability to participate in trials in other countries. It does not, however,remove the related practical difficulties of travel, expense and family/carer disruption

• Early diagnosis – More training for doctors is the obvious suggestion to help increaseawareness of a possible myeloma diagnosis. However, because it is such a rareoccurrence in a lifetime of medical practice, the best option must be continuing medicaleducation and regular reminders of the devastating consequences of a misseddiagnosis, plus research into diagnostics

• Input of patients’ priorities into policy-making – Although patient organisations arenow able to participate in the working of many ethical and regulatory committees, thisinput still needs to spread further in all European countries. Input of patients’ views andpriorities has met with some resistance, but there is plentiful evidence that they can addmaterially to the effectiveness of health authorities’ decisions. The UK government’scommitment to ensuring that patients and the public have a voice in how cancerservices develop was given in its 2011 strategy for improving outcomes in cancer93



Recommendations: Policy

For national healthcare policymakers:

• Develop a clear national cancer plan and within it, clear objectives for diagnosis,treatment and management of myeloma; while taking patients’ quality of life into fullaccount as an essential outcome

• If a national plan is already in place, bring it into full implementation and ensureeffective provision for governance, budget, targets, timelines and review

• Include quality of life indicators in market authorisation dossiers82

• Re-examine national criteria for pricing and reimbursement of orphan drugs toensure that access to them is not restricted for patients on the basis of price

• Work to implement EU recommendations on centres of expertise and Europeanreference networks, especially as they develop in the field of blood cancers

• Work towards uniform treatment guidelines to encourage more uniform access toinnovative treatments, a uniform level of reimbursement and a high level of carethroughout Europe

• Establish or expand patient registries for cancer and specifically myeloma, to gatherevidence on health-related quality of life in addition to basic epidemiological data


• Work to develop knowledge of national and European policies and the expertiseavailable for interaction with national policymakers

• Monitor the establishment of European reference networks and advocate for fullaccount to be take of patients’ needs

• Monitor the transposition by national governments of the EU cross-border healthcaredirective to ensure optimal access to treatments, and call on governments to selectdirect repayment in national mechanisms rather than reimbursement of patientexpenditure

• Advocate for representation within the national health policy-making bodies, withincommittees contributing to authorisation of clinical trials, reimbursement decisionsand health technology assessments

• Advocate for a uniform patient pathway (providing for personalised options), withreimbursement of medications at the same level throughout Europe

• Advocate for a faster drug authorisation process, or a wider access to drugs on thebasis of conditional approval or approval under exceptional circumstances, i.e.before full authorisation

• Advocate for access to clinical trials being conducted in other countries, as this canoffer access to novel drugs or combinations during the time between pivotal studiesand market authorisation



6. The power of patient advocacy in myeloma:making it happen



Patient organisations are now a well-established and recognised partner with all of theprofessional and representative groups concerned with healthcare. They have the knowledgeand commitment to present the patient’s views and to press for attention and improvementwhere it is needed. Across Europe are many examples of their achievements; but also manyother opportunities where greater successes are possible, through advocacy directed towardsopinion-leaders in healthcare or politics, or through information and support to patients andtheir carers. Probably the greatest challenges to patient organisations are those of providing aprofessional service through a volunteer workforce, with limited finance, and of maintainingindependence from other stakeholders.

6. The power of patient advocacy in myeloma: making it happen

Key points

• Patient advocacy exists to:o Promote excellence in myeloma treatment and careo Improve treatment and quality of life for myeloma patientso Provide information and practical support or patients and their carerso Contribute towards, and improve access to research and new treatmentso Contribute patients’ views and experiences in the making of healthcare policy

• Patient organisations depend on patients, carers or other supporters for theiractivities and operation

• Patient groups have very substantial and far reaching effects• Alliances with medical professional organisations give access to their skills and

knowledge, enabling effective interaction with healthcare regulators, payers,politicians and other interested groups

• Raising awareness of myeloma among general practitioners and orthopaedicspecialists for a quicker diagnosis and referral

Advocacy goals

The aim of advocacy in myeloma is to improve, in every way possible, the treatment prospectsand overall quality of life for those affected by the condition. This can be through providinginformation or practical support for patients, contributing and increasing access to researchinto new treatments, or representing patients and advocating for their views and experiencesto be taken into account in healthcare policies. In Europe this is being achieved through theefforts of national and regional patient organisations and those of individual patients,healthcare professionals and corporate supporters.

Myeloma Patients Europe (MPE) is an umbrella network of these patient organisations andsupporters, formed in 2011 by the merger of the former associations, European MyelomaPlatform and Myeloma Euronet.



appenThe aims of Myeloma Patients Europe

• Collaborating on projects to the benefit of the myeloma community*• Exchanging information and best practice• Developing existing patient groups, and encouraging and facilitating the setting up of

new groups• Helping to shape appropriate health-related policies and initiatives on a European

and national level• Ensuring patients across Europe receive timely access to new treatment• Stimulating and promoting patient-centred research and clinical trials• Developing a strong evidence base for the needs and wants of patients, and their

role in research• Providing information, educational and outreach programmes to member groups§§§§

Most of the work of individual patient organisations is carried out by patients, carers or othersupporters, who are volunteers and may be limited by illness or have other commitments. Forthis reason, it is important to develop alliances with other organisations, which give MPE andmany individual patient organisations access to the skills and knowledge of healthcareprofessionals and other experts.

This enables effective and professional interaction with all the many groups and organisationsthat have interests in and influence over what actually is offered to myeloma patients – thehealthcare regulators and reimbursement authorities, politicians, the pharmaceutical industry,the media and through them, the public.

Even within each patient organisation, though, ideally the structure, communication andresponsibilities should be clearly designated in order to achieve effective activities94. Advocacyand participation in professional regulatory and ethics committees demand a thoroughunderstanding of both the scientific and medical aspects of the subject, and the ability toconvey the patients’ perspectives effectively.

What have myeloma patient organisations achieved to date?

A 2012 survey of senior healthcare professionals and pharmaceutical industry executivesfound that European and national patient groups as a whole were well regarded in theircountry95. Half of the healthcare professionals and 42% of the industry executives believedthat their own medical specialty had been prioritised by their government as a result of patientorganisation campaigns95. The same survey revealed that these two groups also regularlyused the websites, online forums and social media sites of patient organisations as a sourceof information95.

In myeloma, one of the most significant single achievements of the patient movement hasbeen in work by several patient organisations in Europe in collaboration with thalidomidevictims’ associations. In the US, thalidomide (Thalomid®) is distributed under a strict RMP(Risk Management Plan) and PPP (Pregnancy Prevention Programme), called STEPS.However, the conditions of the STEPS programme were not appropriate to the EU context andEuropean myeloma patients.

§§§§ e.g. as a partner in research projects with European Myeloma Network, and with EMA and thalidomide victim associations concerning risk management plans and pregnancy prevention programmes for thalidomide,lenalidomide and soon pomalidomide



The collaborating associations signed a common position paper, reflecting the agreement ofall involved on the need for an effective RMP and PPP for the use of thalidomide (Europeanbrand name: Thalidomide Celgene®) for myeloma treatment in Europe, differing in keyrespects from the US STEPS programme96.

In the following years, several meetings with the patient organisations, the victims’associations and the regulators took place at the agency, during which input on the EU coreRMP and PPP for Thalidomide Celgene® and Revlimid®, a thalidomide analogue, was given.The result was that an EU core RMP and PPP were adopted by EMA and implemented withthe marketing authorisation of Revlimid® (2007) and Thalidomide Celgene® (2008). Since then,all EU countries where Revlimid® and Thalidomide Celgene® are marketed follow the coreEuropean version of the RMP and PPP64 .

Patient organisations also have had an important impact on the content and design of clinicaltrials.

Myeloma Patient Groups

Achievements:

• Well regarded, with many experts using websites, online forums and social mediasites of patient groups as a source of information

• Increased overall survival of myeloma patients over the last 10 years by pushingthrough the initiation of a Risk Management Plan (RMP) and Pregnancy PreventionPlan (PPP) in Europe, enabling the authorisation of Revlimid® and ThalidomideCelgene®

• An important influence on the content and design of clinical trials

Challenges:

• Economic constraints• Increasing workload• Maintaining independence from sponsors/supporters• Ensuring a complete and effective service, while relying on patients and volunteers

as a workforce with support from healthcare professionals



Patient organisations’ successes – some more examples

UK: The TAKE 2 Campaign

The Myeloma Diagnosis Pathway is a simple but effective decision-making tool for GPs,developed by Myeloma UK. It is designed to reduce delays in the diagnosis of myeloma byoutlining its signs and symptoms and the investigations to carry out if GPs suspect someonehas myeloma. It has been provided to over 7,000 GPs across the UK, through asking patients,family and friends to take two copies of the Pathway to their GP and encourage two friends todo the same. A film about it was also launched for wider email distribution. GPs have reportedback on how useful they found it. Dr Lynn Jones, a London GP, commented: “I was reallyimpressed by how useful and informative the Myeloma Diagnosis Pathway is. I am now using itin my practice and I encourage my colleagues to do so too”. Myeloma UK is now working tohave it introduced into established GP education and in-house training programmes throughoutthe UK.

Slovakia: Multimedia education course

The Slovak Myeloma Society is a network of myeloma patient groups across Slovakia, whoseaim is to raise the quality of patients’ lives, to help them feel they are not alone with theirproblems, and to work together with family members and relatives. The Society’s Patients’ Clubwas set up to provide information on the diagnosis, treatment and care of people living withmultiple myeloma, and support for their families. A major project has been to publish a simpleCD-based course about myeloma and its treatment for patients, nurses and anyone affected bymyeloma; in Slovak (2007) and English (2011). The course uses colourful slides and animationsto explain the facts in a highly visual and accessible way. It has been presented in local andnational patients’ meetings in Slovakia, and also at European-level patient organisationmeetings and patients’ days in London, Vienna, Brussels and Zurich.

Germany: Setting up new patient support groups

The APMM network of regional myeloma groups in Germany (ArbeitsgemeinschaftPlasmozytom, Multiples Myelom) supports the establishment of new myeloma self-help groupsin regions that do not have one already. It offers help with organising and financing for aPatients’ Day. These are always organised in co-operation with the haematology department ofthe regional Universitätsklinik. Setting up these myeloma groups is welcomed and greatlyrespected by the doctors, and generates their support for the group in the future. In addition,the APMM website is kept up to date at all times with the most recent news about myeloma.

Spain: Multiple initiatives for patient support

AEAL is the Spanish Group for Lymphoma, Myeloma and Leukaemia patients (AEAL), In May2012, it created a subgroup, Mieloma España, at the request of the large number of myelomapatients and their relatives seeking support. In the short time since then, Mieloma España hasalready continued the work for myeloma patients already under way in the parent organisationAEAL. Each year since 2006, AEAL has made up to ten visits to different Spanish towns toraise awareness of myeloma, and participated in the annual Spanish Cancer Patient Groupcongress in Madrid. Online seminars have been held since 2010, to give myeloma patients freeinteractive access to presentations from haematologists; all of which are recorded and availablefrom the AEAL, GEPAC (Grupo Español de Pacientes con Cáncer) and Mieloma Españawebsites. AEAL also developed an online elearning facility called e-Cancer Campus, offering amyeloma education course for patients and carers. Psychological and social support is alsoavailable to patients from AEAL, and as a more practical means of support, the AEAL Apoyaproject has provided orthopaedic beds for myeloma patients with bone problems.



The EFPIA code of practice for relationships with patient

organisations81

• The independence of patient organisations, in terms of their political judgement,policies and activities, shall be assured

• All partnerships between patient organisations and the pharmaceutical industry shall be based on mutual respect, with the views and decisions of each partnerhaving equal value

• The pharmaceutical industry shall not request, nor shall patient organisationsundertake, the promotion of a particular prescription-only medicine

• The objectives and scope of any partnership shall be transparent. Financial and non-financial support provided by the pharmaceutical industry shall always be clearly acknowledged

• The pharmaceutical industry welcomes broad funding of patient organisations frommultiple sources

Challenges faced by myeloma patient organisations

Financial resources:The greatest challenge must be maintaining adequate finances at a time of economicconstraint; offering a service at a time although operating through limited donations or fund-raising. Healthcare systems are experiencing greater pressure than ever before, whilepatients have increased their knowledge and desire to be actively involved in their own care,and are increasingly turning to patients’ associations and voluntary groups for information andreassurance. The net result for patient organisations is an increasing workload and a limited ordecreasing budget.

Maintaining independence:Secondly, the pharmaceutical industry is a valuable supporter of some initiatives of patientorganisations; however patient representatives must maintain their independence and ensurecompletely transparent arrangements with industry sponsors. This principle is safeguarded bythe code of practice for relationships between the industry and patient organisations adoptedby the European Federation of Pharmaceutical Industries and Associations (EFPIA) in 2007and updated in 201198. Member associations of EFPIA (the national pharmaceutical industryassociations) were required to adopt the code into their own national codes of practice byDecember 2011.



Professionalism:The third challenge is the need to ensure professionalism. Many patient organisations arestaffed by volunteers, who may also be patients, and are presented with an extremelydemanding prospect. Their agenda is likely to involve interaction and negotiation with medicalexperts; requiring a high level of knowledge and understanding of a very complex disease.Some organisations would have difficulty in providing individuals able to meet theserequirements and have two options: to enlist the support of medical professional organisationsor to engage in staff training programmes – again, this may be a challenge for funding.

Driving change from West to East

The ultimate aim of the myeloma patient movement in Europe must be to promote excellencein myeloma care, throughout all of its countries and regions. This report has shown manydiscrepancies between the best available care and what is available in practice, particularly inthe newer Eastern EU member states and also in rural regions, where patients need to travellong distances for specialist care.

The European Union is committed to reducing disparities between its regions, as part of itsdrive to strengthening the economic and social conditions across all the member states. Itacknowledges that health is a vital part of those efforts, as a healthier population contributes toeconomic and sustainable growth and makes less demand on healthcare systems99.

Between 2007 and 2013 the EU has also committed over €5 billion to improving healthinfrastructure through the European Regional Development Fund (ERDF) and the EuropeanSocial Fund (ESF). Among current projects relevant to myeloma are almost €78 million for theconstruction of the Centre of Preclinical Research and Technology (CePT) in Warsaw, Poland,which will focus on fundamental research, new diagnostic methods and new therapeuticapproaches; and almost €67 million for upgrading the North Estonia Medical Centre, Tallinn, to offer advanced treatment for oncology and cardiac patients100,101.

Patient movement input to the European Union

At European level, patients’ associations for myeloma (like those for cancer and rare diseases)need to be in active contact with the European Commission’s directorates concerned withhealth and with research; monitoring policy development and making input on patients’ priorityissues.

Also, importantly they should be in contact with relevant members, groups and committees ofthe European Parliament, who can draw the attention of politicians and policymakers to thecritical issues for myeloma in Europe by asking formal questions of the Council andCommission and by arranging discussion forums. Within the Parliament, MEPs have alreadyadopted a declaration calling for improved co-ordination of cancer research102.

Formerly meeting within the cross-party group MEPs against Cancer (MAC), interested MEPsnow participate in the Forum Against Cancer Europe (FACE) initiative of the European CancerPatient Coalition (ECPC). FACE is well aware of the issues relating to rare cancers, but inputspecific to myeloma would be valuable103.



What can patient advocacy do?

• Effective advocacy. This can have very substantial and far-reaching effects. As shownin the examples given above, it can contribute to making myeloma a priority on nationalhealth agendas, and support research programmes

• Provide expert patient advocates, able to present the needs of myeloma patients to allstakeholders in myeloma – healthcare professionals or administrators on a local level, orregional or national policymakers, reimbursement officers, ethics or regulatorycommittee experts, or industry or academic researchers, or the media

• Identify and engage with the key opinion leaders who are receptive to informationfrom the patient organisation and in a position to improve myeloma care

• Provide information, training and personal experience of myeloma. The areaspatient representatives are able to address will depend on the circumstances, cultureand critical issues in their own country. These may be the availability of trials, access tospecialists, reimbursement levels or any of the many diverse aspects of living withmyeloma. Clearly in some countries the patient-doctor and patient-health authorityrelationships are more open to input from a patient representative than in others

• Provide education for all patient advocates. This is a central aim for MPE, which isnow offering national patient organisations training in specialist aspects. Not all patientadvocates will be able to present the most complex aspects e.g. the health economicimplications to health authorities. The ideal would be to train and encourage somepatient advocates to address e.g. clinical trials and research issues, while others focuson regulatory and reimbursement issues e.g. making input to health technologyassessments; while any could speak for the organisation on living with myeloma

• Active and structured collaboration with professional medical associations and

scientific societies. These can add substantially to the resources of patientorganisations. Within these organisations, interaction with individuals, committees andtask forces can elicit expert advice and support, for example in negotiations withregulators and policymakers. Taking part in joint initiatives with professionalorganisations will result in increased recognition of patients’ perspectives

• Share resources from other myeloma associations. Not every patient organisationhas the financial or personnel resources to do everything that it might wish. However, itis always possible to share resources; either in person or by electronic means. Some ofthe associations have already developed large and comprehensive informationresources which could be made available to other associations. The same principle ofworking through others can be applied where an organisation is unable to gainmembership of a committee: it may be possible to reach an understanding with andmake indirect input through an existing member

• Share information and advice. A wide range of other tools can be used by patientorganisations to raise general awareness or to advocate on particular issues, eitherdirectly or by informing key opinion leaders. Again, some of the member associationswithin MPE have well-developed examples that can be shared or adapted, and may wellbe able to share information or advice. They include:

· Websites, with news, expert articles, discussion forums

· Social media sites

· Advocacy events, e.g. roundtable discussions with experts and selected media



· Social events with a local focus and media presence

· Input to medical or social care conferences, e.g. presentations, posters

· Publications – journal or magazine articles

• Develop or share existing programmes for informing healthcare professionals

about myeloma. This is an extremely productive option for patient organisations –particularly in the areas of diagnosis, novel treatments, and psychosocial support.Programmes can be either in group training or individual online resources such as isoffered in the UK by Myeloma Academy*****. At present the resource is tailored forhaematologists and nurses, and will soon also cover GPs and other healthcareprofessionals. It offers best practice guidance, a searchable database on drugs indevelopment, a clinical trials tracker and detailed guidelines and key publications.

• Raising funds. As voluntary organisations dedicated to improving the lives of peoplewith myeloma, their patient organisations depend for their funding on the work ofvolunteers and the generosity of the public. As well as practical fund-raising activities,patient organisations are well placed to raise awareness of their financial needs foractions and campaigns, through their contact with other influential organisations,government bodies and funding sources

***** http://www.myeloma-academy.org.uk/



Recommendations: Patient advocacy

For national patient organisations:

• Identify, make contact with and inform key opinion leaders who are in a position andhave the influence to make a difference to the services available to people withmyeloma. These are among all stakeholders in myeloma: healthcare professionals,administrators on a local level, regional or national policymakers, reimbursementofficers, ethics or regulatory committee experts, industry or academic researchers,media

• Collaborate actively and in a structured way with professional medical associationsand scientific societies to obtain their expert advice and support in negotiations withregulators and policymakers

• Develop the knowledge and abilities of team members within the patientorganisation, drawing on information resources from other national organisations and taking up opportunities for training in effective advocacy and in businessmanagement

• Encourage and train individual patients who have the aptitude to become expertpatient advocates

• Seek opportunities for patient representatives to participate in key policy-making and ethics committees and in the design of clinical trials

• Seek opportunities to raise awareness of myeloma among healthcare professionalsand the public, e.g. through information workshops

• Explore the information and communication resources of other national organisationsand share ideas that could be adapted

For Myeloma Patients Europe:

• Develop access to education in advocacy on specialist aspects of myeloma for thestaff of patient organisations

• Develop a Europe-wide database of information resources and successful advocacyinitiatives, to be available to the patient organisations

• Promote the training resources of the Myeloma Academy to healthcare professionalsin other European countries

• Continue interaction and engagement on key issues with all relevant EU institutions– the Commission’s Directorate General for Health and Consumer Affairs, the HealthWorking Group of the European Parliament’s Committee on Environment, PublicHealth and Food Safety, and individual Members of the European Parliament

7. Acca



7. Achieving excellence in myeloma care – case studies



Achieving excellence in myeloma care – case studies

Germany: Keeping informed about clinical trials

In the Universitätsklinikum Heidelberg, the Physician and patient – hand in hand project is runjointly with the regional myeloma patient organisation. In daily practice, this means that theleader of the self-help group comes to the patient, whenever and wherever needed; called bythe clinical personnel responsible, usually the doctor. Through this practice, the group leader isaccompanying the patient step by step, explaining to him/her exactly what is going on. In thisway, it is easier for the patient to accept the treatments and actions which are necessary, andit also encourages them to participate in the clinical trials offered.

The Netherlands: Finding out what patients really need

The Netherlands Myeloma and Waldenström association CMWP* provides information for bothpatients and carers including a regularly updated patients' handbook, support groups,newsletters and contact persons. At the CMWP annual symposium, speakers present thelatest developments in medical treatment as well as emotional aspects.

Working with researchers at Maastricht University, it is currently developing a survey of CMWPmembers, followed by focus group discussions, to define what aspects of myeloma care in theNetherlands they believe to be most important in future research, and what could be improved.The results will be analysed together with the findings from a literature survey, and the reportwill present a list of priorities that can be used to inform future clinical, education or informationinitiatives of the association, especially in future research.

UK: The Myeloma Academy™ and the Myeloma Nurse Programme™

The Myeloma Academy is an online resource designed to meet the educational needs ofhealthcare professionals involved in the treatment and care of myeloma patients. It containsdesignated educational materials for both hospital doctors and nurses, including best practiceguides, position papers, tutorials and challenging cases. The Myeloma Academy is currentlyaccessed by over 300 healthcare professionals across the UK, including nurses, consultanthaematologists and clinical scientists.

Within the Academy's resources, the Nurse Best Practice Guides aim to enhance nurseknowledge, inform nursing practice and support nurses to deliver the highest quality oftreatment and care to myeloma patients and their families. The Guides have recently beenaccredited by the Royal College of Nursing (RCN). The Myeloma Nurse Programme is anetwork of Myeloma Nurse Champions in UK myeloma centres, with access to resources forrole development, education of colleagues, implementation of best practice and developinglocal patient services. Further information is on: http://www.myeloma-academy.org.uk/



Acknowledgements

MPE Writing Group:

Dr Faith Davies, member of the Faculty at the Institute of Cancer Research (ICR), London andConsultant Haematologist at The Royal Marsden, London.

Greetje Goossens, Myeloma Patients Europe

Peter Lens, Myeloma Patients Europe

Professor Heinz Ludwig, Head of the Department of Medicine and Medical Oncology, andDirector of the Myeloma Reference Centre at the Wilhelminen Hospital in Vienna, Austria.

Ananda Plate, Myeloma Patients Europe

Lia Van Ginneken-Noordman, (Contactgroep Myeloom en Waldenström Patiënten (CMWP))

and …

For their valuable input:

Wioletta Niznik (Weber Shandwick)

Jude Leitch (Myeloma UK)

For their stories of living with myeloma: For examples and best practices:

Johan Creemers, Belgium Alfonso Aguaron, Spain

Judy Dewinter, UK Miroslav Hrianka, Slovakia

Greetje Goossens, Belgium Christa Kolbe-Geipert, Germany

Christa Kolbe-Geipert, Germany Kate Morgan, UK

Mika Peltovaara, Finland Lia Van Ginneken-Noordman, The Netherlands

Stewart McRobert, UK Dr Tiny Van Merode, The Netherlands

Report writer:

Dr Vivienne Kendall, UK


CPMW

Anonymus

Sponsors:

We are very grateful for the generous support of the following groups and organisations:


About MPE

What is Myeloma Patients Europe?

Myeloma Patients Europe (MPE) is an umbrella organisation of multiple myeloma patientgroups and associations from across Europe. MPE was formed following the merger in 2011 ofthe European Myeloma Platform and Myeloma Euronet.

MPE is registered as a non-profit organisation under Belgian law and its office is located inBrussels.

To meet its aims and in addition to its membership, MPE works directly with healthcareprofessionals, reimbursement authorities, regulators, politicians, pharmaceutical companies,the media and anyone involved in the ‘myeloma community’.

What does MPE do?

MPE has a number of broad aims including:

• Collaborating on projects to the benefit of the myeloma community

• Exchanging information and best practice

• Developing existing patient groups and encouraging and facilitating the setting up of new groups

• Helping to shape appropriate health- related policies and initiatives on a European and national level

• Ensuring patients across Europe receive timely access to new treatment

• Stimulating and promoting patient- centred research and clinical trials

• Developing a strong evidence base for the needs and wants of patients and their role in research

• Providing information, educational and outreach programmes to member groups

How is MPE structured and who is involved?

MPE acts as an umbrella organisation for existing local and national myeloma associationsand its members come from nearly 30 different countries.

There are two types of members:

Full members such as patients’ groups or associations from European countries.

Associate members such as individual patients or healthcare professionals as well ascorporations willing to make donations or to help in other ways.

A Board is in place and has responsibility for setting strategy as well as for governance andaccountability. The Board is multidisciplinary but is constituted to have at least 50% of itsmembers as being patients or care-givers.

MPE’s Board members are all volunteers who are supported by the staff at the Brussels office,as well as by an expert Medical Advisory Committee.

To become a member of MPE please download the application form fromwww.myelomapatientseurope.org or request one at [email protected]



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