EHC Workshop on Economics and HTA’s for EU Member
Organisations
September 20th, 2014
Keith Tolley
Director
Tolley Health Economics Ltd
keith@tolleyhealtheconomics.com
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Disclaimer – my opinions
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These are economically challenging
times for health care payers!
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Pressures on the payers…
 Drug expenditures continue to rise
 New drugs are rarely cost saving
 New drugs for previously untreated conditions
 Ageing, obesity, etc
 Growing patient awareness
 Budgets for health care becoming even more constrained
 Always been constrained - Economic austerity of last few years
meaning becoming even more constrained
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In economically challenged times new innovative
therapies need to represent good value for
money!
 Need to provide the patient, health service and societal
benefits at a cost that payers are willing to pay
 The more an unmet need is met the more payers may
be willing to pay!
 This willingness to pay can be expected to vary across
healthcare payers in Europe (depending on budgets
and health care priorities)
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Innovation is about addressing an
unmet need!
“Neither newness
nor effectiveness
separately or
together constitute
innovation”
Source: Morgan et al. 2008, Open Medicine 2 (1):E4-7
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GETTING MARKETING
AUTHORISATION
GETTING MARKET
ACCESS
Health
Benefit
Clinical
Benefit
RISK
COST
Market access controls
Not all drugs that get marketing authorisation get market access.
CONTROLLED by:
 ………..restrictions on the price for public reimbursement
 ……….limiting the level of reimbursement
 ……….controlling market access through Health Technology
Assessment (HTA) – UK situation
Health Technology Assessment
 Health Technology Assessment (HTA) is “a form of policy
research that systematically examines the short-and longterm consequences, in terms of health and resource use,
of the application of a health technology” (Henshall et al.,
1997)
 HTA has been used as a tool across many countries to
help national and regional authorities and payers assess
the added value new pharmaceuticals bring:
 To determine reimbursement and/or price at market access
 To inform clinical guidance/prescribing
 HTA in Europe is co-ordinated through organisations such
as EUnetHTA
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National decision makers and local payers
Decision
makers:
National
reimbursement
and HTA bodies
e.g. NICE in UK,
TLV in Sweden,
IQWiG in
Germany,
AHTAPol in
Poland
Local Payers:
Local payers are health
authorities and hospitals
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What are health care decision
makers and payers willing to
pay for?
Not just newness, or
new mechanism of
action alone, or new
formulation!
But……
The health care benefits a
drug brings:
• Clinical benefits
• Survival improvements
• Quality of life (QoL)
improvements
Healthcare resource
savings:
• Reductions in hospital
inpatient use
• Significant
improvements in the
quality of care
Wider Societal benefits:
• Quality of life benefits
for family members and
caregivers
• Improved economic and
social returns
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What does a payer want to know?
 What offers best value – payers want value based
assessments?
 What can we afford?
 Perspective and what is included within “value” is
important and varies by country/type of payer:
 In Sweden a societal perspective is adopted
 In UK a healthcare and social service perspective is adopted, but
moving more ‘societal’
 Regional payers (Spain, Italy) concerned with budget impact, cost
offsets
 Local payers focussed on affordability in every country
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Assessments of cost-effectiveness
 The QALY is growing in use around Europe as the
‘standard’ measure of the health benefits (and hence
value) of new pharmaceutical interventions
 Long established in UK and several other European
countries
 More recently seen to be adopted in European
countries relatively new to the use of HTA e.g. Estonia,
Croatia
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UK Benchmark for decisions:
incremental cost per QALY gained (Historical)
Probability of rejection on
grounds of cost
ineffectiveness
A = <£20,000 per
QALY gained:
-Considered an
efficient use of
resources
B = >£30,000 per
QALY gained
-Would need special
circumstances to
accept
Increasing cost/QALY (log scale)
Source: Rawlins and Culyer, BMJ 2005;329:224-227
Questions of value in haemophilia
Possible questions an HTA organisation could ask:
 Which approach to the treatment of haemophilia A with inhibitors
offers the best value?
 Is the reimbursement of prophylactic use of factor VIII better value than
treating bleeds on-demand in haemophilia A or B?
 Do new recombinant factor VIII products offer good value for money for
use in prophylaxis compared to existing recombinant products such
that they should be reimbursed?
 What additional benefits do they bring e.g. less frequent administration?
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Published studies
 Most attention given to cost-effectiveness of prophylaxis
versus on-demand factor VIII treatment (more recent
attention on cost-effectiveness of inhibitors treatment)
 Early health economic studies (90’s) did not have QALY as
the outcome measure
 Recent systematic review found 7 cost per QALY studies
(CUA’s):
 Two were assessments of treatments for inhibitors
 Five were assessments prophylaxis v on-demand
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Cost-utility analyses
Study
Study design and countries
Utility measure used
Ekert et al, 2001
CUA of recombinant factor VIIa (Novo7) v usual
care for haemophilia with inhibitors
Before and after NovoSeven.
Single hospital - Australia
Generic EQ 5D
Knight et al, 2003
Alternative on-demand regimens for haemophilia
A with inhibitors
Decision model - UK
Generic EQ 5D
Miners et al. 2002 & Miners 2009
Prophylaxis vs on-demand in severe haemophlia
Markov model – UK
Generic EQ 5D
Lippert et al, 2005
Prophylaxis vs on-demand in severe haemophlia
Cross sectional study patients
aged 14+ years – Ger, Swe, UK,
Neth
Generic SF 6D
Risebrough et al 2008
Tailored prophylaxis, vs primary prophylaxis vs
on-demand in young children 0-6 years
Markov model - Canada
Direct Standard gamble in
general public
Colombo et al 2011
Prophylaxis vs on-demand in severe haemophlia
Markov model - Italy
Generic EQ 5D
Farrugia et al 2013
Prophylaxis vs on-demand in severe haemophlia
Markov model – US, UK, Swe
Generic EQ 5D/SF 6D
Key results
Study
Treatment/patients
considered
Incremental
costs
QALYs
gained in a
cohort
Cost/QALY
Result
Miners
2009
Lifetime treatment with
prophylaxis vs ondemand for severe
haemophilia in UK
(70 yrs follow-up)
£214,000
5.63
£38,000 per QALY
gained for primary
prophylaxis
Colombo,
2011
Lifetime treatment with
prophylaxis vs ondemand for severe
haemophilia in Italy
(70yrs follow-up)
€787,000
16.03
€40,000 per QALY
gained for primary
prophylaxis
Ferrugia
2013
Lifetime treatment with
prophylaxis vs ondemand for severe
haemophilia in 4
countries
-£280,000 (UK)
9.69
-
SEK 5.3 million
(Swe)
10.99
SEK 485,000 per
QALY gained
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Importance of uncertainty!
 Cost-effectiveness prophylaxis vs on-demand is sensitive to:
 Perspective adopted
 Dosage and cost of factor VIII assumed
 Probability of inhibitor with prophylaxis vs on-demand
 Time horizon and discounting
 Quality of life estimated for prophylaxis vs. on-demand:
Source: Noone et al, 2013
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HTA and payer questions of value of a
new treatment
 What are the patient reported benefits – how many
QALYs can be gained for the population?
 How high is the incremental cost per QALY gained –
what is the opportunity cost of funding the new
treatment?
 How robust, how uncertain are we of the costs and
benefit – how much risk do we want to take?
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Summary – HTA and payer perspective
 HTA is a supportive tool for decision making, based on
demonstration of value.
 It takes a population rather than individual perspective, but
what’s important in that perspective can vary.
 Can expect closer HTA scrutiny of haemophilia in the
future: focussed on what value do new haemophilia
treatments bring, are they worth paying a premium price?
 What value for longer half life, less frequent administration?
 Will payers be willing to pay more for this?
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