1
Assessment of Pharmaceuticals to Inform Reimbursement Decisions
in Portugal
Vasco A. J. Maria
INFARMED – Autoridade Nacional do Medicamento e Produtos de Saúde
Portugal
VIII Curso de evaluación e selección de medicamentos
Palma de Mallorca, 7 de Mayo de 2010
PORTUGAL
10 Million inhabitants
National Health ServiceUniversal
comprehensive free of charge
covers all the population
Other Health Systems (Civil Servants, and other) responsible for 3 millions
All the population has the right to health to be delivered through NHS
Assessment of pharmaceuticals to inform
reimbursement decisions in Portugal
Assessment of pharmaceuticals to inform
reimbursement decisions in Portugal
Total Health Expenditure as % of GDP - 2006
Despesa em Saúde em % do PIB (2006)
10,2
10,0
7,0
9,6
8,3
11,0
10,5
9,5
8,1
7,1
9,0
7,3
9,7
6,2
7,3
8,4
9,1
8,5
9,9
0,0 2,0 4,0 6,0 8,0 10,0 12,0
Austria
Belgium
Czech Republic
Denmark
Finland
France
Germany
Greece
Hungary
Ireland
Italy
Luxembourg
Netherlands
Poland
Portugal
Slovak Republic
Spain
Sw eden
United Kingdom
Fonte: OECD HEALTH DATA 2009, Nov. 09
Assessment of pharmaceuticals to inform
reimbursement decisions in Portugal
Total Medicines Expenditure as % of GDP - 2006
Despesa Total de Medicamentos em % do PIB (2006)
2,6
2,2
2,2
2,2
1,8
1,8
1,8
1,7
1,6
1,6
1,4
1,2
1,2
0,8
1,6
0 0,5 1 1,5 2 2,5 3
Hungary
Greece
Portugal
Slovak Republic
France
Italy
Spain
Poland
Czech Republic
Germany
Austria
Finland
Sw eden
Denmark
Belgium
Fonte: OECD HEALTH DATA 2009, Nov. 09
Assessment of pharmaceuticals to inform
reimbursement decisions in Portugal
Total Medicines Expenditure as % of Total Health Expenditure - 2006
Despesa Total de Medicamentos em % de despesa com Saúde (2006)
8,5
31,8
29,7
27,2
22,8
22,7
21,8
21,7
19,9
16,3
15,9
14,8
14,3
14,2
13,7
13,3
0 5 10 15 20 25 30 35
Denmark
Hungary
Slovak Republic
Poland
Czech Republic
Greece
Portugal
Spain
Luxembourg
France
Belgium
Germany
Finland
Ireland
Sw eden
Austria
Fonte: OECD HEALTH DATA 2009, Nov. 09
Assessment of pharmaceuticals to inform
reimbursement decisions in Portugal
Total Medicines Expenditure per capita - 2006
Consumo de Medicamentos per capita nos países da OCDE - 2006
151
221
226
288
397
412
471
504
517
530
536
542
551
568
587
599
644
710
0 100 200 300 400 500 600 700 800
Poland
Czech Republic
Slovak Republic
Hungary
Portugal
Denmark
Finland
Spain
Greece
Austria
Sweden
Norway
Germany
Italy
Switzerland
Belgium
France
Iceland
US$Fonte: OECD HEALTH DATA 2009, Nov. 09
• HospitalsNHS or third payer is responsible for all the expenses with in-patient consumed medicines
• PharmaciesNHS or third payer is responsible for all or part of the expenses with consumed medicines
on the purchasing act the consumer does not pay or pay only a part of medicine’s price
Assessment of pharmaceuticals to inform
reimbursement decisions in Portugal
Assessment of pharmaceuticals to inform
reimbursement decisions in Portugal
Source: INFARMED, I.P.; IMS Health; ACSS
Medicines Expenditure Growth2000-2008
-5%
0%
5%
10%
15%
20%
25%
30%
20002001
20022003
20042005
20062007
2008
Gro
wth
rat
e
Total Market (outpatient) NHS Market (outpatient)NHS expenditure (outpatient) Hospital Market
Assessment of pharmaceuticals to inform
reimbursement decisions in Portugal
0
200
400
600
800
1000
1200
1400
1600
Mill
ion E
uro
s
Source: INFARMED 2009
Expenditure by NHS 1996-2008
Causes of growing drug expenditure:
Increase in population of elderly residents
Increase incidence and duration of chronic diseases
Continuing development of health technologies
Increase in health expectations by patients and
society
Higher prices (R&D costs, attrition rates)
Assessment of pharmaceuticals to inform
reimbursement decisions in Portugal
Health Technology Assessment
What do we measure in HTA?
Relative effectivenessAdditional benefit in clinical practice of the new medicine in comparison with
alternative therapies (EBM)
Cost-effectivenessComparison of two relevant alternative
therapies (innovatorversus standard treatment)
Need to:
Promote the development of new tools to provide support on
decision making process - Economic Evaluation Studies of
Medicines (1998)
Create guidelines to implement good practices on the
execution and evaluation of Economic Evaluation Studies of
Medicines - Guidelines (1999)
Create levels of quality to graduate the clinical evidence used
for assessment of pharmaceuticals to inform reimbursement
decisions (2000)
Assessment of pharmaceuticals to inform
reimbursement decisions in Portugal
Clinical
Assessment
Economic
Assessment
Relative effectiveness
added therapeutic value
Cost-effectiveness
“value for money”
Relative effectiveness and cost-effectiveness evaluation for reimbursement decision – ambulatory and hospital
Assessment of pharmaceuticals to inform
reimbursement decisions in Portugal
Evidence based report to
support the decision
• Applicants
Marketing Authorization Holders (MAH)
(submit all necessary evidence)
• INFARMED
Administrative assistants
Pharmacists
Doctors
Health Economists
Executive Board of INFARMED
• Minister of Health
Assessment
Decision
Assessment of pharmaceuticals to inform
reimbursement decisions in Portugal
10 external experts coming from universities and hospitals (doctors and health economists) *
Internal staff
– 6 economists
– 4 pharmacists
– 2 administrative assistants
Team:
* Names and CV are available at INFARMED webpage
Assessment of pharmaceuticals to inform
reimbursement decisions in Portugal
Identify if the medicine is for an unmet need
Identify the relative effectiveness/added therapeutic value
Extent to which an intervention does more good than harm compared to one or more intervention alternatives for achieving the desired results when provided under the usual circumstances of health care practice.
(Pharmaceutical Forum)
Clinical Assessment
Assessment of pharmaceuticals to inform
reimbursement decisions in Portugal
Identify the value for money
- Comparative price analysis for generics and medicines without added therapeutic value using equivalent daily treatment
- Economic evaluation study for medicines that fulfil an unmet need or with added therapeutic value (innovative medicines) in order to identify the cost-effectiveness ratio
Identify the implications for NHS budget
Economic Assessment
Assessment of pharmaceuticals to inform
reimbursement decisions in Portugal
Application by MAH
Assessment Procedure
Application form (/CFT form)/Scientific information/Additional data
Clinical assessment
Preliminary Report (structured format)(critical appraisal and n. of patients)
Final Report
TherapeuticEquivalence
Added Therapeutic
Value
Inferior Therapeutic
Value
Additional data
PVH ≥ comp.
Price comparative analysis
Economic advantage
PositiveDecision
MEES [185d]
NegativeDecision
Therapeuticequivalence
Added therapeutic
value
Inferior therapeutic
value
PVH < comp.
Assessment Procedure
Decision(positive/negative)
MEES/clinical report
Preliminary Report
Final Report
Additional data
Economic Assessment
Assessment Procedure
Decision (positive/negative)
Reports (clinicians/economists/pharmacists)– Peer discussion and adoption by consensus
Consolidated Final Report
Summary report (to be publish in the webpage) (revised by assessors)
Proposal for CFT monitoring procedures)
Final proposal for Decision by MB
Assessment Procedure
Final proposal for Decision by MB
Negative Decision
Information to MAH
10d for counter argumentation
Reanalysis
Final negative decision
Positive Decision
Contract (2 copies) to be signed by MAH
Signed contract(MAH + MB)
Information to MAH/HospitalsPublication in webpage
Assessment Procedure
Agreement for 2 years (to be renewed)
Additional demonstration of ATVCeiling for NHS expenditure with the medicine in
all public hospitals Estimation of the number of patients to be treatedMonitoring mechanisms Consequences if ceiling is surpassedMaximum price considered to be adequate
Agreement and Signed Contract
Field of Application
NCE reimbursed since 2001
22 23
41
24
12
23
1520 17
327 6
14
2001 2002 2003 2004 2005 2006 2007 2008 2009 Jan-Mar2010
(*)
Ambulatory Hospitalar
(*) 3 under agreement negotiation
Medicines for Hospital usePositive Decision Negative decision
Abatacept Lenalidomida Ácido lipoico
Agalsidase Alfa Leuprorrelina Bevacizumab
Anidulafungina Levofolinato dissódico
Carbetocina
Ambrisentam Maraviroc Oxibato de sódio
Capecitabina Mecassermina Ziconotida
Cefeminox Panitumumab
Cladribina Raltegravir
Darunavir Ranibizumab
Dasatinib Sitaxentano
Efavirenz + emtricitabina + tenofovir
Sorafenib
Etravirina Tacrolímus
Fibrinogénio humano + Trombina humana
Temsirolímus
Gadoversetamida Tenofovir
Medicines for Hospital use
Year of Signature Number
2007 2
2008 7
2009 14
2010 (Jan-Mar) 3
Total 26
Number of agreements (contracts)
Critical Issues/Constraints
•Selection of comparators
•Uncertainty (lack of information)
•Orphan drugs (rare diseases)
•Target-population (subgroups)
•Budget impact
•Time pressure
•Pressure from pharmaceutical industry
Basic Principles:
Scientific independence Conflicts of interest
Robustness of assessment procedure Best experts in the field
Evidence based
Peer discussion and decision by consensus
Transparency Clear definition of criteria
Publication
Segregation between assessment and decision
Assessment of pharmaceuticals to inform
reimbursement decisions in Portugal
Gracias por su atención