Operations & Expertise

Vlerick Healthcare Conference27-10-2016

Improving access to medicines by tilting the balance towards societal priority setting

Dr Chris Van Hul

Staff Study & Representation

Independent Mutualities

2

TitleLet’s start with a comparison..

The eighties (of last century)

Comfortable Budgetary margins

Blockbusters based on great volumes

Hardly any societal comments on huge

benefit margins for drugs

Much innovation but few studies about

cost-effectivity

Hardly any commercial interest in

development of new therapies for small

patient populations

All are targeting the same

pharmacological groups:

benzodiazepines, antidepressants,

cephalosporines, hypolipidemiants,..

A rather flexible legislation about study

protocols

now

Budgetary restrictions with closed

budget for drugs

Value for money is a major point of

attention

Cost of research increased ++ (much

more stringent legislation being one of

the causes)

Blockbusters: shift towards drugs for

orphan diseases, more valorising than

large quantities

Antibiotics: few in the pipeline

3

TitleWhat resulted, in those times, in:

a price assessment without a real critical

discussion about cost-effectivity and global

budgetary impact

An absence of thorough reflection

(ministry/payers/society) about priorities

No urgent need for cost- containment contracts

4

TitleTimes they are a changing..

Budgetary constraints

an avalanche of extremely expensive drugs (cancer, mucoviscidosis,

other rare diseases,..), with others to follow (personalised medicine..)

A more critical attitude of care givers, payers, patients: HTA, EBM,

comparison between countries, comparison with non-medicinal therapies

Large public debate

Value

Uncertainty

Budget impact

Making choices

Key questions for the assessment of the value of a new drug

1. Is there a medical, therapeutical or societal need?

2. Is there a willingness to pay from public funding for this specific therapy?

3. Is there a willingness to pay for this specific drug?

4. Is there a willingness to pay more for this drug than for the best alternative?

5. How much do we want to pay (price and reimbursement)?

6

TitleThis debate was however not really “public”

Colloque singulier between 3 partners (“the usual

suspects”):

Government

Industry

Payers/insurers

No real input from the consumers (patients and/or citizens)

Although the government (democratically mandated) and the payer (the

mutualities) can claim to be the rightful representors of the consumers,

there is growing insight that a direct input from the consumers offers a

plus and that there are different estimations of value

7

TitleAn answer to austerity: managed entry schemes

>100 contracts in Belgium

Orphan diseases, oncolytica, hepatitis C,..

About uncertainty (patient population etc.)

And even much more about cost- containment and

budget impact

Hardly about the other priorities of the citizens

8

TitlePrioritisation in times of austerity

Managed entrance schemes, but what else?

Prioritisation: from offer to demand

How to decide on the highest needs?

Prioritisation as an answer to waiting lists and cost-

containment

Dealing with uncertainty by making choices

How to involve all the relevant actors

9

TitleUK: a tradition of stakeholders implication:

NICE and the Citizen councils

Reimbursement in the UK:

1. Pharmaceutical Price Regulation Scheme

2. NICE

3. Cancer Drugs Fund

10

TitleNICE and the Citizen councils

NICE appraisal of new drugs: evaluation of

Clinical effectiveness (health effects)

and

Cost effectiveness (value for money)

based on:

Clinical trials and peer reviewed research

Economic evidence (cost for NHS)

Views of clinicians, patients and other

stakeholders

11

TitleNICE and the Citizen councils

QALY, thresholds

>£30,000: needs a strong case to be accepted

“NICE appraisals may not capture the full value of

a drug” (Postnote House of Parliament January 2015). They

may not sufficiently value drugs

that treat very severe conditions

or

which have wider societal benefits (to patients, carers,

other public service users)

Conclusion: need for Value based assessment

and value based payment (VBA,VBP)

12

TitleNICE and the Citizen councils

9 criteria Severity of underlying disease

Unmet need

Significant innovation

Wider societal benefits

Disadvantaged populations

Children

End-of-life treatments

Cancer treatments

Rare diseases

Stakeholder persuasion

13

TitleCitizens Council

Panel of 30 members of the public that largely

reflect the demographic characteristics of the UK

Recruited by an independent organisation

3 years maximum

Since 2002

18 reports published

14

TitleCitizens Council:

the inspiration for Citizens implication

Report 1 (2002): “What should NICE take into

account when making decisions about clinical

need?”

Report 4 (2004): premium prices for drugs to treat

very rare diseases?

Report 10 (2008): Qaly’s and severity of illness

Report 15 (2011): assessment of future costs and

health benefits

Report 17 (2014): trade-off between equity vs

efficiency

15

Title

16

TitleCancer Drugs Fund

Introduced in 2010 to enable patients to access

drugs not available on the NHS (not recommended

as being cost effective or not yet appraised)

Intended as an interim measure until a wider value-

based appraisal system would be in place.

Based on the assumption that there is a societal

consensus about higher pricing for cancer

medication (cfr the £20,000/30,000/QALY

acceptability)

17

TitleCancer Drugs Fund

95,000 patients reimbursed but costs rising from 50 million to 200, 280

and finally 340 million (2015/16)

Huge societal debate about the future of the CDF

Breakthrough Breast Cancer: extension across the UK

CRUK (Cancer Research UK’s): greater public awareness, equal access to

innovative radiotherapy

Some cancer specialist argue that the money spent in the Fund might

generate better outcomes if spent for prevention, early diagnosis,

radiotherapy, surgery..

New approach since 29/07/2016

12 week public consultation

Further engagement with key stakeholders including patient groups and industry

18

TitleSocietal preferences for the funding of

medicines: a study that raises questions

“Do the criteria used by NICE (and the Cancer Drugs

Fund) for accepting higher incremental cost-

effectiveness ratios for some medicines really reflect

societal preferences for NHS resource allocation?"

Cross-sectional survey of 4118 adults in UKHealth Economics 2013

Warren Linley and Dyfrig Hughes

19

TitleCross-sectional survey of 4118 adults

UK 2013

Web-based questionnaire to broadly UK-

representative sample of citizens

“Allocate fixed funds between different patient and

disease types reflecting nine specific prioritisation

criteria”

20

TitleCross-sectional survey

UK 2013

Only 3 criteria clearly supported:

Severity

Unmet needs

Innovation (if offering substantial health benefits)

• No compelling evidence for 3 other prioritisation criteria, if

considered separately (disadvantaged populations, children, end-

of-life)

• Life saving ok, life extending perhaps

21

TitleCross-sectional survey

UK 2013

9 criteria (cfr NICE) Severity of underlying disease

Unmet need

Significant innovation

Wider societal benefits

Disadvantaged populations

Children

End-of-life treatments

Cancer treatments

Rare diseases

Stakeholders persuasion

22

TitleValue based assessment: the sequel

New evaluation criteria: propositions:

Wider societal impacts (calculating absolute

shortfall)

Burden of illness( calculating proportional

shortfall)

“Evidence from surveys on what society values is

limited and mixed. What little evidence there is

suggests greater public support for placing more

value on QALYs for those with a higher burden of

illness, than for those needing EOL treatments.”

23

TitleThe Belgian Context

A joint action of NIHDI, KBF and KCE

KBF: 4 methodological workshops (since June

2013) , 3 citizen labs and an expert meeting

KCE: 5 reports since 2008

NIHDI: Report on societal dialogue “50 years

NIHDI”

24

TitleKing Baudouin Foundation

25

TitleKBF: results citizens lab

Less prolongation of life, more quality of life

Less technocratic, more human

Less offer induced innovation, more demand

driven

More integrated cure responding to the global

needs of the patient

Less waste and more effectiveness

Presentation Template

PPT 2010

basisvragen

Presentation Template PPT 2010

2008 2010 2012 2014 2016

28

Study 2014-06

Methods for ranking

therapeutic needs

29

TitleFaster acces to drugs by identifying real

“Unmet Medical Needs”

The unmet medical needs procedures foresees in the possibility

to obtain early temporary reimbusement (ETR) for products

which treat an unmet medical need and which are included in a

medical need or compassionate use programme approved by the

FAMHP.

A cohort decision implies ETR for an unmet medical need.

Requests for a cohort decision can be submitted by a

pharmaceutical company, the Minister of Health or the College of

Medical Directors at the RIZIV / INAMI.

The cohort decision is taken by the College of Medical Directors,

following the advice of the ‘commission for advice on temporary

compensation for the cost of a pharmaceutical product’

(CAIT / CATT).

The CAIT / CATT assesses whether the product targets an unmet

medical need, based on the list of unmet medical needs definedby the General

Council of the NIHDI KCE report 272

32

Title

34

TitleMulti-criteria decision analysis for the

appraisal of medical needs

KCE 272 (28/06/2016)

The list of stakeholders who can submit proposals for inclusion

on the unmet medical needs list should be expanded to include

patients, healthcare providers and other professionals.

Budgets for innovative treatments for high healthcare needs

should not be limited to pharmaceutical innovations but be

expanded to other types of innovations in the healthcare sector.

Presentation Template PPT 2010

36

TitleAris Angelos & Panis Kanavos

PharmacoEconomics (january 2016) (2016)

Value-Based Assessment of New Medical

Technologies: Towards a Robust Methodological

Framework for the Application of Multiple Criteria

Decision Analysis in the Context of Health Technology

Assessment

(London School of Economics)

37

TitleValue tree hierarchies and data sources

using a multiple criteria decision analysis framework

for value assessment

38

TitleSocietal Conclusionsviews on Nice, Cancer

drug fund and

Citizens are an important stakeholder in the decision making process

The best way to involve the citizen in concreto in the decisional

process is still a “work in progress”

In Belgium a joint action was organised in the last few years

Multi-criteria decision analysis that takes into account the

preferences of the citizens is a promising tool for making objective

choices

The Unmet Medical Need” procedure has to become demand-driven

39

TitleA few suggestions

Give the patient associations an advising role in

more instances

Involve them in the “Institute of the Future” that

will determine national health objectives

Develop a methodology to question the citizens

On a regular basis

On a large scale

e.g. by joining a questionnaire at the Health Interrogation

Survey (every 3 years)