VIII Curso de evaluación e selección de medicamentos
Palma de Mallorca, 7 de Mayo de 2010
Assessment of Pharmaceuticals to
Inform Reimbursement Decisions
in Portugal
Vasco A. J. Maria
INFARMED – Autoridade Nacional do
Medicamento e Produtos de Saúde
1
Portugal
Assessment of pharmaceuticals to inform
reimbursement decisions in Portugal
PORTUGAL
10 Million inhabitants
National Health Service
Universal
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
Total Health Expenditure as % of GDP - 2006
Despesa em Saúde em % do PIB (2006)
8,5
United Kingdom
Sw eden
9,1
8,4
Spain
Slovak Republic
7,3
9,9
Portugal
Poland
6,2
9,7
Netherlands
Luxembourg
7,3
9,0
Italy
Ireland
7,1
Hungary
8,1
Greece
9,5
Germany
10,5
France
11,0
Finland
8,3
Denmark
9,6
Czech Republic
7,0
Belgium
10,0
Austria
10,2
0,0
2,0
Fonte: OECD HEALTH DATA 2009, Nov. 09
4,0
6,0
8,0
10,0
12,0
Assessment of pharmaceuticals to inform
reimbursement decisions in Portugal
Total Medicines Expenditure as % of GDP - 2006
Despesa Total de Medicam entos em % do PIB (2006)
Belgium
1,6
0,8
Denmark
Sw eden
1,2
Finland
1,2
Austria
1,4
Germany
1,6
Czech Republic
1,6
Poland
1,7
Spain
1,8
Italy
1,8
France
1,8
Slovak Republic
2,2
Portugal
2,2
Greece
2,2
Hungary
2,6
0
0,5
Fonte: OECD HEALTH DATA 2009, Nov. 09
1
1,5
2
2,5
3
Assessment of pharmaceuticals to inform
reimbursement decisions in Portugal
Total Medicines Expenditure as % of Total Health Expenditure - 2006
Despesa Total de Medicam entos em % de despesa com Saúde (2006)
Austria
13,3
Sw eden
13,7
Ireland
14,2
Finland
14,3
Germany
14,8
Belgium
15,9
France
16,3
Luxembourg
19,9
Spain
21,7
Portugal
21,8
Greece
22,7
Czech Republic
22,8
Poland
27,2
Slovak Republic
29,7
Hungary
31,8
Denmark
8,5
0
5
Fonte: OECD HEALTH DATA 2009, Nov. 09
10
15
20
25
30
35
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
710
Iceland
644
France
599
Belgium
587
Sw itzerland
568
Italy
551
Germ any
542
Norw ay
536
Sw eden
530
Austria
517
Greece
504
Spain
471
Finland
412
Denm ark
397
Portugal
288
Hungary
Slovak Republic
226
Czech Republic
221
151
Poland
0
100
Fonte: OECD HEALTH DATA 2009, Nov. 09
200
300
400
US$
500
600
700
800
Assessment of pharmaceuticals to inform
reimbursement decisions in Portugal
• Hospitals
NHS or third payer is responsible for all the expenses with inpatient consumed medicines
• Pharmacies
NHS 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
Medicines Expenditure Growth
2000-2008
30%
25%
Growth rate
20%
15%
10%
5%
0%
-5%
00
20
01
20
02
20
03
20
Total Market (outpatient)
NHS expenditure (outpatient)
Source: INFARMED, I.P.; IMS Health; ACSS
04
20
05
20
06
20
07
20
NHS Market (outpatient)
Hospital Market
08
20
Assessment of pharmaceuticals to inform
reimbursement decisions in Portugal
Expenditure by NHS 1996-2008
1600
Million Euros
1400
1200
1000
800
600
400
200
19
96
19
97
19
98
19
99
20
00
20
01
20
02
20
03
20
04
20
05
20
06
20
07
20
08
0
Source: INFARMED 2009
Assessment of pharmaceuticals to inform
reimbursement decisions in Portugal
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)
Health Technology Assessment
What do we measure in HTA?
Relative effectiveness
Additional benefit in clinical practice of
the new medicine in comparison with
alternative therapies (EBM)
Cost-effectiveness
Comparison of two relevant alternative
therapies (innovator
versus standard treatment)
Assessment of pharmaceuticals to inform
reimbursement decisions in Portugal
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
Relative effectiveness and cost-effectiveness evaluation for reimbursement
decision – ambulatory and hospital
Clinical
Economic
Assessment
Assessment
Relative effectiveness
Cost-effectiveness
added therapeutic value
“value for money”
Evidence based report to
support the decision
Assessment of pharmaceuticals to inform
reimbursement decisions in Portugal
• Applicants
Marketing Authorization Holders (MAH)
(submit all necessary evidence)
• INFARMED
Administrative assistants
Pharmacists
Doctors
Assessment
Health Economists
Executive Board of INFARMED
Decision
• Minister of Health
Assessment of pharmaceuticals to inform
reimbursement decisions in Portugal
Team:

10 external experts coming from universities and hospitals
(doctors and health economists) *

Internal staff
–
6 economists
–
4 pharmacists
–
2 administrative assistants
* Names and CV are available at INFARMED webpage
Assessment of pharmaceuticals to inform
reimbursement decisions in Portugal
Clinical Assessment
 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)
Assessment of pharmaceuticals to inform
reimbursement decisions in Portugal
Economic Assessment
 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
Assessment Procedure
Application by MAH
Application form (/CFT form)/Scientific
information/Additional data
Clinical
assessment
Preliminary Report (structured format)
Additional
data
(critical appraisal and n. of patients)
Final Report
Inferior
Therapeutic
Value
Therapeutic
Equivalence
Added
Therapeutic
Value
Assessment Procedure
Inferior
therapeutic
value
Therapeutic
equivalence
Added
therapeutic
value
Price comparative analysis
PVH < comp.
Economic
advantage
Negative
Decision
Positive
Decision
PVH ≥ comp.
MEES [185d]
Assessment Procedure
MEES/clinical
report
Economic Assessment
Preliminary
Report
Additional
data
Final Report
Decision
(positive/negative)
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
Positive
Decision
Contract (2 copies) to be
signed by MAH
Signed contract
(MAH + MB)
Reanalysis
Final negative
decision
Information to MAH/Hospitals
Publication in webpage
Agreement and Signed Contract
Agreement for 2 years (to be renewed)
 Additional demonstration of ATV
 Ceiling for NHS expenditure with the medicine in
all public hospitals
 Estimation of the number of patients to be treated
 Monitoring mechanisms
 Consequences if ceiling is surpassed
 Maximum price considered to be adequate
Field of Application
NCE reimbursed since 2001
41
22
24
23
23
20
17
15
12
14
7
3
2
2001
2002
2003
2004
2005
Ambulatory
(*) 3 under agreement negotiation
2006
2007
2008
Hospitalar
2009
6
JanMar
2010
(*)
Medicines for Hospital use
Positive 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
Number of agreements (contracts)
Year of Signature
Number
2007
2
2008
7
2009
14
2010 (Jan-Mar)
3
Total
26
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
Assessment of pharmaceuticals to inform
reimbursement decisions in Portugal
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
Gracias por su atención
vasco.maria@infarmed.pt