CHE Seminar presentation by Professor Lise Rochaix 6th February 2014

1. ‘Economic Evaluation of Health
Technologies in France: from
Theory to Practice
Lise Rochaix, Ph.D
Professor at the economics department of AMU (Aix-Marseille
University) and member of GREQAM (Groupe de Recherches
Quantitatives d’Aix-Marseille), former member of HAS board.
CHE Seminar Series, February 6th, 2014

2. The author declares
having no conflict of interest
2

3. In their 1983 paper on Regional allocation of health
care resources in the UK and France, Alan Maynard
and Ann Ludbrook regarded France as ‘le mauvais
élève’ (the last of the class) for health care expenditure
management*. The purpose of the presentation is to
assess whether this still holds today. To address the
question, the seminar will draw on the recent
experience of the French National Health Board (HAS
–Haute Autorité de Santé-), which was given legal
remits in 2008 and 2012 to carry out economic
assessments of health care technologies and
interventions. The obstacles encountered in the process
of setting up the new efficiency advices on pricing for
new technologies will be reviewed, as well the
prospects for further developments in the use of
economic analysis for decision-making in health care
in France.
* Social policy and administration, vol. 17, number 3
3

4. 01
The context
4

5. The ‘open-ended’ funding approach
Rather than define priorities and make explicit choices, France has,
over the past 30 years:
1 – expected that growth would generate enough value to
finance increasing needs
2 – assumed that waste could be reduced and would match
the growing financial needs ;
3 – reduced other public expenditure (such as defence) for
the benefit of the health care sector
4 – borrowed from future generations by running fast
increasing deficits …
These strategies can no longer be adopted in order to
postpone (or shy away) from choices and priority setting 5

6. The OECD 2010 shared diagnostic
While no definite answer has been given to the macroefficiency’ question of ‘how much is enough’… with
variations in the percentage of GDP spent on health care
All EU countries converge on the need to:
make limits on health care spending more explicit …
and more binding
make the best use of available resources (allocative and
productive efficiency)
Define priorities increasingly based on costeffectiveness studies
6

7. OECD assessment for France
The French health care system shows:
•
a generous cover (‘over the basic coverage
performance’ indicator)
•
A better than EU average information on quality and
prices
•
An ability to set priorities …
Coupled with:
•
Limited stringency in the budget constraint
•
Little ability to control prices billed by providers
•
Restricted levers for insurers (basic coverage)
7

8. How to reap efficiency gains?
The new set of policy indicators provides guidance
8

9. The ‘true’ obstacles to change
• Reluctance to make clear choices (eg the ‘gatekeeping saga’)
which makes implementation difficult
• Lack of recognition of the need to prioritize at national level,
due to doctors’ legal obligation of means
• Fragmentation of professions’ representatives, strong
corporatism and medical power in decision-making
• Exacerbation of the value of freedom of choice compared to
other objectives (equity, efficiency)
• Economics associated with cost containment and rejection of
utilitarianism and Qalys
• Little use of evidence to support public policy definition
• Lack of adequate information systems on which to base
9
contracting and incentives

10. What is needed:
• Increased governement accountability to Parliament through
greater respect of the national health insurance annual
expenditure target (=> making the constraint on resources
explicit and credible)
• Systematic and robust evaluation of public policy
• Prioritization to ‘verticalize’ the 100 public health priorities
• Use full evidence when assessing health care strategies,
including economics
10

11. 02
Can HAS help?
A case study on drug
pricing
11

12. HAS: an integrated approach
1
3
2
Assess Medical
Technologies
What does HAS assess?
•Medicinal products
Recommend Healthcare
Strategies
What type of
recommendations
does HAS provide?
•Medical devices
•Clinical Practice Guidelines
•Health technologies and
medical procedures
Act to improve healthcare
quality
What does HAS do?
•Public health
recommendations
•Certify medical practice
evaluations
•Chronic diseases
management
•Healthcare safety guidelines
Integrate medical,
organisational
and economic
factors
•Accredit HCOs
Define quality
evaluation
criteria for
action
•Provide information and
mediation on adverse
events
•Certify medical
information provision
HAS ensures high-quality healthcare for all
12

13. PARLIAMENT
Ministry of Health
Votes law and
oversees implementation
Advice/public health policy
Pricing
Committee Advice/
price
(CEPS)
Haute Autorité
de Sante
Advice/reimbursement
Indicative
spending
targets
!
National health insurance funds
(compulsory and voluntary)
Negotiation with
Unions of health professionals
13

14. In 2011, OECD countries spent on average USD$ 500 per
capita for outpatient pharmaceuticals, or 1.6% of GDP, with
wide variations
Pharmaceutical expenditure per capita
Pharmaceutical expenditure as a share of GDP
701
673
648
648
632
627
615
587
541
531
525
520
500
487
483
479
469
445
443
Total (no
breakdown)
Prescribed
436
433
419
373
Over-thecounter
357
325
319
284
272
266
259
178
1000
800
600
400
200
2.1
United States
Canada
Greece ¹
Japan
Ireland ¹
Germany
Belgium
France
Australia
Hungary
Switzerland
Slovak Republic ¹
Austria
Spain
Italy ¹
OECD31
Netherlands ¹
Portugal
Korea
Sweden
Finland
Slovenia
Iceland
Czech Republic
Norway
Luxembourg
Poland
New Zealand
Estonia
Denmark
Mexico ¹
Chile
985
0
USD PPP
1. Includes medical non-durables.
Source: OECD Health Statistics 2013 , http://dx.doi.org/10.1787/health-data-en.
1.9
2.6
1.9
1.6
1.6
1.6
1.8
1.4
2.6
1.0
2.2
1.3
1.6
1.5
1.5
1.1
1.8
1.5
1.1
Public
1.2
1.7
Private
1.4
1.5
0.6
0.6
1.5
1.0
1.3
0.7
1.7
0.9
0
1
% GDP
2
3
4
14

15. Generic market shares vary
widely across OECD countries
%
80
76
75
73
72
70
Value
Volume
60
51
44
40
42
41
36
36
35
28
35
34
34
30
28
24
20
29
25
23
17
19
15
18
17
23
21
18
16
15
12
6
9
9
16
9
8
3
0
1. Reimbursed pharmaceutical market. 2. Community pharmacy market
15
Source: OECD Health Statistics 2013

16. Pricing and reimbursement decisions for
a new technology
HTA
guidance
CEPS
Economic Committee for
Healthcare Products
L
I
S
T
I
N
G
Price
Decision
16
16

17. The French drug pricing system
• Primarily based on added therapeutic value assessement (ATB)
by HAS transparency committee (CT)
• Two types of instruments to contain drug expenditure
– Price setting together with volume contracting, using clawback
payments in case of overshooting
– Restrictions on target population
• A hybrid between
– an international reference pricing system for drugs with
major to moderate added therapeutic benefit (ATB=1,2,3)
– An additional therapeutic assessment system for ATB =4
– An internal reference pricing system (using price of national
comparators) for ATB =5
17

18. Initial listing: From HAS guidance to CEPS pricing
Dimensions
Clinical aspects
• clinical efficacy
Criteria
Thera.
Benefit
‘TB’
Results
Insufficient
Sufficient
• clinical effectiveness
• relative effectiveness
Other aspects
No ATB(5)
• target population
Added
Therap.
value
• impact on public health
‘ATB’
• disease characteristics
Minor
ATVB (4)
Reimbursement
only if price inferior
to comparators
Price may be
higher than
comparators
High to
moderate
ATB(1,2,3)
HTA: HAS Guidance
No reimbursement
‘European’ Price
P
R
I
C
I
N
G
Decision: Ministry
Pricing: 18
Economic Committee18

19. CEPS pricing rules
Balancing objectives
Optimal use of limited healthcare resources
Public health promotion
Innovation support
Decision tree /Added Therapeutic Value (ATB)
• Major to moderate ATB (1 to 3): eligible for faster access at a
European price (Price notification in place of negotiation)
• Minor ATB (4): individual firm price/volume agreement
• No ATB (5): price lower than comparators by Law
Additional criteria
• Competitors’ prices in same therapeutic indication
• Forecast or recorded sales volumes
• Expected and/or actual conditions of use
19

20. From therapeutic added benefit …
The French drug pricing system relies on an assessment of the
added therapeutic benefit by the transparency committee, based
on individual clinical benefits
– The added value to society (social value) is not addressed
per se
– The other price determinants (among which, industrial
considerations) are taken into account by the French
pricing committee (CEPS) but there is no decision
traceability (confidential agreements)
20

21. … to collective added value
To be truly ‘value based’, pricing has to rely on the explicit and
quantitative assessment of all the individual and collective value
determinants
=> Additional elements must be documented, beyond the
measurement of therapeutic added benefit (relative
effectiveness):
• Efficiency gains measurement (both expected and effective)
• Organizational impacts
• Social values
=> The steps towards including economics into HAS advice
21

22. Step 1: The 2008 Law
• Role of economic analysis strengthened for public health
and clinical practice guidelines
Screening, type 2 diabetes, …
• Cost-effectiveness analysis for class reassessment (at most
within 5 years) with possible impact on price
Statines, stents, ..
• For some technologies, full HTAs (inc. ethics and social
values)
Growth hormones for non deficient children
 50 economic assessments produced as well as
methodological guidelines for economic assessment
 But no economic analysis at first listing
22

23. Methods
http://www.hassante.fr/portail/jcms/r_1499251/fr/choi
x-methodologiques-pour-l-evaluationeconomique-a-la-has?xtmc=&xtcr=1
23
23

24. Methodological guides
1 – Methods for public consultation
2 – Choices in methods for economic
evaluation at HAS
3 – Integration of ethical dimensions in HAS
guidance
4 – joint diagnostic tests and treatments (work
in progress)
24

25. The challenges for economics at first listing
• Time frame for first listing advice:
=> Can we do it in 90 days?
• Added therapeutic benefit not assessed
• => Can we do it in parallel?
• Price information availability:
- Link between individual drug value and price is
difficult to establish as price negotiation at firm level
- Actual prices differ from facial price through rebates
 Can simulations over price ranges be used?
• Strategic interaction between HAS and CEPS:
=> How far can HAS go in documenting all dimensions of
a price decision without locking CEPS in its negotiation?
25

26. Step 2 – The 2012 Law
The 2012 Financing law for social security (LFSS 2012) strengthens
the role of HAS in documenting the collective added value
(efficiency) at 1st listing
– HAS’ existing committee (economics and public health
evaluation committee – CEESP) set up in 2008 has now the same
legal status as HAS medical committees for drugs (transparency
committee - CT) and medical devices (CNEDIMTS)
– From 22 to 33 members (various profiles: economics,
ethics/philosophy, other social sciences, patient representatives,
public health specialists, GPs and specialists)
– CEESP is in charge of establishing and disseminating public
health and economic recommendations as well as efficiency
assessments for 1st listings and reassessments
26

27. Step 3: The Oct. 2nd, 2012 Decree
• The decree specifies that from Oct. 3rd, 2013, firms will have
to submit economic dossiers, based on HAS guidelines, both
for first listing and reassessments (1 year for implementation)
• Two conditions:
i) Only applies for ‘innovative’ drugs and medical devices (ie
for which the firm claims an added therapeutic benefit
(ATB/ASMR) of level 1 to 3 (=>European prices)
ii) The forecasted expenditure must have a significant impact
on the health care budget
• CEESP will produce and publish efficiency guidance for the
pricing committee on expected efficiency within 90 days, in
parallel to the medical guidance
27

28. Added Therapeutic Benefit (ATB)
granted by transparency committee
ASMR/ATB
2008
2009
2010
2011
2012
I-II
7
10
4
1
6
III
5
8
8
2
10
% I-III
13
18
14
5
16
IV
17
20
20
20
22
V
48
65
46
29
58
Total number
of advice
75
100
85
58
99
28
28

29. ATB claimed / granted for drugs in 2012
• Added Therapeutic Benefit (ATB) claimed by firms for
drugs:
– ASMR I : 5
– ASMR II : 12
– ASMR III : 20
• ATB given by HAS transparency committee:
– ASMR I : 0
– ASMR II : 6
– ASMR III : 10
=> Potentially between 30 and 40 efficiency advice
produced by HAS for drugs (less for medical devices)
29

30. What is meant by the second
condition: ‘significant impact’?
• A three way concertation between CEPS/HAS/Industry
representatives (October 2012 – sept. 2013)
• Simulations carried out by HAS to define an entry criterion for a
CE analysis to be provided by firms at first listing
• A joint HAS/CEPS decision (sept. 2013):
– New drugs with expected gross turnover (for a full year) over
20 million € (for all indications) must have a CE analysis on
which HAS will produce an expected efficiency advice
– The firm also briefly documents the expected impact of the
new technology on the health care system
– HAS Board may decide to require a CE analysis if expected
impact is high, even with a turnover is below 20 million
30

31. 31

32. From Nov. 3rd, 2013
• CEESP efficiency advice to CEPS for 1st listings
– methodological quality of the economic part of the
application submitted by companies (budget impact
only considered if CE analysis also available
– expected efficiency analysis (based on Incremental
cost-effectiveness ratio – ICER -) together with an
assessment of the robustness of the ratio (sensitiviy
analyses)
– Additional economic data may be requested in postlisting studies
• Full economic analysis at the time of re-assessment
– ICER calculation
– other price determinants (ethical, sociological, …)
32
32

33. 33

34. HAS assessment process
Economic assessment
CEESP
CEPS
Clinical assessment
CT
CNEDIMTS
34

35. 35

36. 36

37. The 90 days economic evaluation stages
1. Dossier submission
2. Checks / administrative completeness and methodological
adequacy
3. Internal analysis at HAS
4. Optional : HAS request for additional data or
clarifications
5. Draft advice
6. CEESP validation
7. Advice sent to company and pricing committee
8. Contradictory phase + price negotiation
9. Final advice published on website
37

38. Challenges ahead
•
Producing guidance on both clinical and economic dimensions
within 90 days
•
Developing early advice to identify relevant data collection for
economic assessment
•
Ensuring complementarity between clinical and economic
dimensions by clarifying respective contributions
•
Identifying important societal dimensions at first listing through a
check list to ensure full analysis at time of reassessment
•
Encouraging use of economics for value assessment (not short
term cost-control) and for all health interventions (not just drugs)
•
Taking part in European initiatives (EunetHTA) to encourage
mutualisation of resources
38

39. Conclusion
•
HAS’ experience with quantifying the individual documented
and expected magnitude of the technology benefit is of relevance
when considering value based pricing
•
The more recent contribution of non-medical disciplines
(economics, sociology, ..) to assessing the collective added value
of technology benefit, in comparison to other strategies, is also
relevant
•
Strengthening the link between HAS guidance (based on both
dimensions) and P&R pricing policy may contribute towards a
more transparent assessment of collective value, provided
economics is not used as a rationing tool
39