Patient Rights in Research

and Treatment

Martinelli Vittorio San Raffaele Hospital, Institute of Experimental Neurology

and University Vita-Salute San Raffaele. Milan, Italy

Disclosures  I received compensatory honoraria for lectures from:

–  Bayer –  Biogen-Idec –  Merck Serono –  Teva Ph –  Novartis –  Genzyme

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The voice and hope of MS people:

A new effective cure for MS?

From Bench to Bed Research: The Patient Clinical World 21

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Gross,C.P. Ann Intern Med. 2002

Recruitment in Clinical Trials

• Rigorous regulatory processes

• Stringent enrolment criteria

• More complex trials

• Lots of trials

Higher n. of pts/ Trial duration

50% Recruitment

failure 24

Aims of our study Challenging Recruitment

Need of in-depth understanding of the perceived and current barriers

No research in MS patients

Observational study

To find out strategies to overcome recruitment challenges

To assess MS patients´ attitude towards RCT

To identify factors affecting participation from patients’

perspective

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MAIN AIMS OF RCTs*:

•  Clinical Relapses

•  EDSS worsening

•  New MRI lesions

•  Progression of brain atrophy 26

The evaluation of:

*Randomised  Controlled  Trials  

MAIN AIMS OF RCTs

PRO But  eventually,  do  you  feel  be:er  ,  the  same  or    worse?  

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Part I : Methods Questionnaires

To understand factors affecting MS patients’ willingness to participate in a RCT

Enrolment Problems Questionnaire (ENPQ)

Sections on:

demographics, knowledge about RCTs, patient’s level of involvement in clinical decision-making, previous involvement in RCTs, willingness to participate in a general RCT, patient’s consideration on the reasons of CCSVI overspread, impact of different facilitators/barriers on participation in RCTs, suggestions on how to increase participation.

Open, closed (fixed alternative and multiple choice)

questions and graded response

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ATTITUDE TO PARTICIPATE

IN RCT Patient - Physician Relationship

Welfare state

Clinical trial already proposed

Already participated to a clinical trial

Disease state

Possible solutions

Participation modalities

Demographics Negative factors Positive factors

Diagnosis Disability

Degree Time from diagnosis

Sex Marital Status

City Cohabitation

Occupational Status Job

Incomes Educational Degree

Age

Knowledge of RCT

A1

A15

F + 1 F + 16 F - 1 F - 8

C6

C1

ATTITUDE TO PARTICIPATE

IN RCT Patient - Physician Relationship

Welfare state

Clinical trial already proposed

Already participated to a clinical trial

Disease state

Possible solutions

Participation modalities Participation modalities

Demographics Negative factors Positive factors

Diagnosis Disability

Degree Time from diagnosis

Sex Marital Status

City Cohabitation

Occupational Status Job Job

Incomes Incomes Educational Degree

Age Age

Knowledge of RCT

A1 A1

A15 A15

F + 1 F + 1 F + 16 F + 16 F - 1 F - 1 F - 8 F - 8

C6 C6

C1 C1

Methods

Know

ledge of R

CT

(6 items)

Negative Factors (8 items)

Positive Factors (16 items)

Supportive Factors

(15 items)

Overall about half of our MS patients were willing to consider

participation in a RCT

Strongest positive predictors:

•  Older age, •  Not having children, •  Progressive MS, •  Previous participation

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Results

Result I: Priority map – negative factors Factors that most influence

patients not to join a trial

1.  The risk of side effects 2.  Negative effects on private life 3.  Burden on daily activities 4.  Effects on family

To feel like a guinea pig

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Result I: Priority map – solutions

1.  Comprehensive  informaAon  on  all  the  experimental  and  approved  drugs  

2.  Contact  details  in  case  of  side  effects/doubts    

3.  Update  on  trial/informaAon  4.  More  Ame  to  explain  the  study  5.  Knowing  the  allocaAon  arm  

Solutions to increase participation

• Proposal  from  different  physician  • Logo/Acronym  

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Improve  successful,  effecAve,  long  standing  paAent–physician  

relaAonship  to  increase  support  to  pa-ents  (telephone  contact,  more  

Ame)  

Provide comprehensive information to patients and their families

(of trial-treatments-allocation)

The Use of online

Platforms

Strategies to overcome recruitment

challenges

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What  is  the  most  important  factor  preven-ng  people  with  MS  from  joining  a  Clinical  Trial?  1.  The  risk  of  side  effects  2.  Effects  on  family  3.  Effects  on  job  4.  Social  expense  5.  Feeling  like  a  guinea  pig  6.  Knowing  that  MS  Neurologists  receive  a  payment  

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0 20 40 60 80%

74%

8%

8%

0%

8%

2%

1. The risk of side effects

2. Effects on family

3. Effects on job

4. Social expense

5. Feeling like a guinea pig

6. Knowing that MS Neurologists receive a payment

MS patients from joining a Clinical Trial?What is the most important factor preventing people with

What  do  you  think  is  the  most  important  factor  to  increase  the  par-cipa-on  of  people  with  MS  in  Clinical  Trials?  

1.  Proposal  from  a  different  physician.  2.  Comprehensive  informaAon  on  all  experimental  and  approved  

drugs.  3.  Audio/video  support  .  4.  Payment  .  5.  Knowing    which  arm  they  were  allocated  to.  6.  Being  sure  they  would  receive  a  be:er  treatment.  

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0 10 20 30 40 50 60%

2%

54%

4%

2%

2%

36%

1. Proposal from a different physician.

2. Comprehensive information on all experimental....

3. Audio/video support .

4. Payment .

5. Knowing which arm they were allocated to.

6. Being sure they would receive a better treatment.

increase patients’ participation in Clinical Trials?What do you think is the most important factor to

Result I: Priority map – solutions

1.  Comprehensive information on all the experimental and approved drugs

2.  Contact details in case of side effects/doubts

3.  Update on trial/information 4.  More time to explain the

study 5.  Knowing the allocation arm

Solutions to increase participation

• Proposal from different physician • Logo/Acronym

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Altruistic consideration

(in ALL patients)

to help other suffering people

to contribute towards furthering medical

knowledge greater chance of a beneficial cure

Individualism (in willing patients)

the only way to be treated

by an expert to get a new agent

Boosting

Hindering

To identify factors affecting

participation from patients’

perspective Burden

(in ALL patients )

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v  concerns about safety/ side effects,

v  the burden of a trial on the family/private life,

v  uncertainty of receiving an effective treatment

Altruistic consideration

(in ALL patients)

Individualism (in willing patients)

Boosting

To identify factors affecting

participation from patients’

perspective Burden

(in ALL patients )

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Hindering

Improve successful, effective, long standing patient–physician relationship to increase

support to patients (telephone contact, more time)

Provide comprehensive information to patients and their families

(of trial-treatments-allocation)

The Use of online Platforms

Strategies to overcome recruitment

challenges

41

The voice and hope of MS people:

A new effective cure for MS?

From Bench to Bed Research

The Patient Clinical World 42

MS?  

CSF  

MRI  

Disease  Course  

SIGNS  

•  Monophasic presentation •  RR •  Progressive course

• Optic nerve • Spinal cord • Brainstem/Cereb

Diagnostic workup in case of suspected MS

The  doctor  

•  They  believe  they  had    done  a  good  job!  •  …but  they  are  Aware  of  their  limits  and  of  those  of  the  medical  science!   44

The Neurologists:

THE  COMMUNICATION  OF    MULTIPLE  SCLEROSIS  DIAGNOSIS:    

THE  PATIENTS’  PERSPECTIVE  Maria  Josè  Messina,  Gloria  Dalla  Costa,  Giancarlo  Comi  and  

Vi:orio  MarAnelli  

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To  communicate  the  diagnosis  of  MS  is  a  difficult  task:  •  It  affects  young  paAents  •  it’s  a  chronic  disease  •  It  has  an  unknown  evoluAon  and  response  to  the  treatments    To  communicate  the  diagnosis  of  MS  is  a  necessary  task:  •  Pts  demand  the  truth:  90%  of  MS  pts  express  the  preference  of  a  clear  diagnosis  as  soon  it  

is  defined  (  Messina  et  al  2015)  •  Pts  demand  the  truth  as  soon  as  possible:  a  delay  in  the  communicaAon  may  reduce  

confidence  between  paAent  and  doctor  (Papathanasopoulos  et  al  2005)  To  communicate  the  diagnosis  of  MS  is  an  important  task:  •  An  effecAve  communicaAon  increase  the  acceptability  of  the  diagnosis  

•  An  effecAve  communicaAon  increase  the  compliance    

The  communica-on  of  the  diagnosis:      the  pa-ents’  perspec-ve    

Patients characteristics Satisfaction rate

The  communica-on  of  the  diagnosis:      The  pa-ents’  perspec-ve    

Highly satisfied Quite satisfied Poorly satisfied

Factors  influencing    pa-ents’  sa-sfac-on  

Informa-on  Asymmetry    Between  Pa-ent  and  Neurologists  

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AIM  of  the  Treatment:    Absence  of  Disease  AcAvity  

No  MRI  Ac-vity    

No  Clinical  Relapse  

No  EDSS  Worsening  

No  Brain  

Atrophy  

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The  AIM  of  The  PaAent  

 Absence  of  Disease  AcAvity    +    «wellbeing!»  

No  MRI  AcAvity  MRI    

No  Clinical  Relapse  

No    EDSS  Worsening  

No  Brain  Atrophy  

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Feeling well Feeling better

A better Communication… to boost “Resilience”

Through  a  Mul-disciplinary  Approach    we  could  offer  an  empowerment  process,    

to  enhance  paAent  Quality  of  Life  and  indirectly    improve  adherence  to  treatments   53

Resilience: The ability to make realistic plans and take the steps necessary to overcome

stress and adversities, without giving up

Factors influencing patient-doctor rapport

Dalla Costa G et al, Neurology 2014

Pa-ent–doctor  rapport  has  a  direct  influence  on  health  outcomes  

EffecAve  paAent–doctor  communicaAon  has  been  associated  with  mulAple  benefits:  

–  greater  ability  to  collect  informaAon  –  be:er  understanding  of  medical  condiAons    –  be:er  cooperaAon  with  treatment  plans  –  greater  paAent  saAsfacAon  

Solari A et al, Multiple Sclerosis 2010 55

IFN  β  1a    30  mcg  

IFN  β  1b  

IFN  β  1a    22  mcg  

Gla-ramer  Acetate  

IFN  β  1a    44  mcg  

Natalizumab  

Mitoxantrone  

Dime-lfumarate  

Fingolimod  

First  Treatment  

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Teriflunomide  

Risk  Propensity  

Risks  Benefits  

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Risk  Propensity  

Risks  Benefits    

MTX  

FTY  

GA   TERI  

BG10  

IFN ß

CYC  

NAT  

MP   GA IFN ß

RTX

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 Mul-centre    Observa-onal      Study    

on  the  Percep-on  and  Propensity  for  Risk    in  Italian  MS  Pa-ents    Star-ng  a  Treatment  

What  is  the  Goal?  

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Today,  people  with  CIS/MS  are  looking  for:  

Reliable and valid information on MS

A better understanding of MS and treatments

A more active role in coping with the disease

Better communication with their healthcare providers and friends

A booklet for patients with a recent MS diagnosis

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Clinical  prac-ces  •  Be:er  knowledge  

•  Be:er  communicaAon  

•  Be:er  cooperaAon  

•  Higher  benefits  

•  Lower  adverse  effects  62

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«…  partnering  with  pa-ents  is  about  a  

fundamental  shic  in  the  power  structure  in  

healthcare  and  a  renewed  focus  on  the  core  

mission  of  health  system»  

Give        VOICE      to  the  PaAents!  •  Veritas  •  ObjecAvity  •  Interest  •  Competence  and  CommunicaAon  •  Empathy  

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Medical  Evidence  

Clinician    ExperAse  

PaAent  Goals    &  Concerns  

A Solari CRNI 2013

Informed Medical Decisions

Conclusions In  a  paAent-­‐centered  health  model  it  is  imperaAve  to  obtain  paAents’  opinions  and  to  determine  strategies  that  can  be  put  in  place,  to  overcome  problems  and  

facilitate  a  successful  treatment    

MS  paAents  who  decide  to  start  a  treatment  must  be  posiAvely  influenced  by  personalized  informaAon  and  a  conAnuous  support.  Checking  frequently  clinical  and  MRI  findings  during  follow  up  should  define  the  changing  benefit  /  risk  profile  

of  the  paAent  over  Ame  and  consequent  decisions  to  make  

Increasing  knowledge    Providing  more  comprehensive  informaAon  to  paAents  and  their  families  

Assuring  a  successful,  effecAve,  long  standing  paAent-­‐physician  relaAonship  

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MS  PaAent  Summit  -­‐  Rome    

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Friday, May 22nd, 2015 Summit Moderator: Kaz Aston (UK)  08:30  

 

08:40  

09:20  

10:00  

10.40  

Welcome & Introduction - Antonella Moretti (AISM, It) & Anna Chiara Rossi (Novartis, It)  

Plenary session  

•  Patient rights in research and treatment - Dr Vittorio Martinelli (It), Aliki Vrienniou (Gr)  

•  Influencing the direction of healthcare decision-making - Mary Baker (UK)  

•  The ‘Expert’ Patient - Jean Hardiman-Smith (UK)  

Panel discussion  

11.15   Break  11.30   Breakout sessions  

•  Patient rights in research and treatment (co-facilitator - Michele Messmer (It))  

•  Influencing the direction of healthcare decision-making (co-facilitator - Silvia Traversa (It))  

•  The ‘Expert’ Patient (co-facilitator - Federica Balzani (It))  12.30   MS World Café  

Best Practice Sharing Fair for Patient Advocates  

13.00   Buffet Lunch  14:00   Breakout sessions (continued)  15:00   Breakout group feedback to plenary  15:55   Closing Remarks – Antonella Moretti (AISM, It) & Anna Chiara Rossi (Novartis, It)  16.00   Meeting Close  

Raising  standards;  The  voice  of    people  with  MS