This presentation addressed core issues in the challenge of evaluating technologies that treat rare diseases. It draws together information about current economic thinking and practices that most affect decisions, offering suggestions as to where and how the patient's input might be most important and influential.

1. Evaluating Highly Specialised
Technologies – An Economist’s View
Jon Sussex
Genetic Alliance Patient Group Workshop
London • 28 November 2013

2. Agenda
1. Cost effectiveness and outcomes
2. Problems evaluating HSTs
3. Factors driving yes/no decisions
4. A value framework for rare disease
treatments?
5. Maximising patient / carer impact
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3. Cost Effectiveness
+ cost
x
x

- effect
x

- cost
+ effect

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4. Patient-reported Outcome
Measures
•
Patients’ own assessment of their health – purposefully
subjective
•
Many well-validated instruments exist that are reliable,
sensitive and widely used
•
Simple to complete; quick to analyse
•
Repeated “snap shots” of health (e.g. before and after
treatment) can provide a clear picture of changes in health
•
Disease specific PROMs: more question items/response
options; focussed on a specific aspect of health
•
Generic PROMs: measure health-related quality of life
generally. Enable comparisons of health across
conditions/health services, e.g. EQ-5D and SF-36
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5. EQ-5D
Please indicate which statements best describe your own health state today. Tick
one box for each group of statements.
Mobility
I have no problems in walking about
I have some problems in walking about
I am confined to bed



Self-Care
I have no problems with self-care
I have some problems washing or dressing myself
I am unable to wash or dress myself



Usual Activities
I have no problems with performing my usual activities
(e.g. work, study, housework, family or leisure activities)
I have some problems with performing my usual activities
I am unable to perform my usual activities



Pain/Discomfort
I have no pain or discomfort
I have moderate pain or discomfort
I have extreme pain or discomfort



Anxiety/Depression
I am not anxious or depressed
I am moderately anxious or depressed
I am extremely anxious or depressed
© 2013 EuroQol Group




6. Measuring
quality of
life using
EQ-5D
© 2013 EuroQol Group

7. Problems Evaluating
Medicines for Rare Diseases
•
Costs not much lower than for other medicines
•
Patents last no longer than for other medicines
– but OMP* legislation helps offset that
•
Small patient numbers mean low sales volumes
and hence high prices
•
Small patient numbers mean longer and more
complex trials yielding more uncertain evidence
*orphan medicinal products
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8. Mean R&D Costs per Successful New
Molecular Entity by Year of Study
Publication (US$2011m)
2,000
1,500
1,000
500
2012
2011
2010
2008
2006
2004
2003
2001
1999
1997
1995
1993
1991
1989
1987
1985
1983
1981
‐
1979
Estimates of mean costs per approved drug (US$m, in 2011prices)
2,500
Source: Mestre-Ferrandiz et
al. 2012
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9. The Commercial Imperative
€
Revenue
minus
production
costs
Launch
R&D costs
Patent
expiry
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Years

10. Accepting Uncertain Evidence
•
If payer is risk neutral, and the state should be,
then no problem in principle
•
But in practice regulators are risk averse
•
And are much more risk averse than sufferers
from severe diseases and their carers
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11. NICE’s Standard Criteria
(weights not revealed)
•
Incremental cost effectiveness ratio (ICER) £/QALY
•
Severity of illness (Rawlins et al, 2010)
•
Stakeholder insights (Rawlins et al, 2010; NICE, 2008)
•
End of life treatments (Rawlins et al, 2010; NICE, 2008)
•
Disadvantaged populations (Rawlins et al, 2010; NICE,
•
Children given “benefit of the doubt” (Rawlins et al, 2010)
2008)
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12. Impact of ICER Ranking on NICE
Recommendations (Dakin et al, 2013)
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£500,000
£100,000
£70,000
£45,500
£50,000
£60,000
£37,500
£40,000
£45,000
£35,000
£32,500
£30,000
£27,500
£20,000
£22,500
£25,000
£17,500
£15,000
£12,500
£10,000
£7,500
£5,000
£2,500
£0
Decisions with high ICERs are more likely to be rejected, but
there are many exceptions

13. Predicted Probability of NICE Rejection at Different
ICER Values – Holding All Other Variables at Mean
Levels (Dakin et al, 2013)
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14. NICE HST Interim Process and
Methods
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15. Who Gives Special Consideration
to Medicines for Rare Diseases?
NICE – England and Wales
Yes – Interim HST process
(previously AGNSS). Also Cancer
Drugs Fund
SMC – Scotland
No – But Rare Conditions Fund
PBAC - Australia
Life Saving Drugs Program and “rule
of rescue” though not about rarity
per se
GB-A - Germany
< €1million exempt from economic
evaluation
Other countries with formal
No
evaluation systems (Canada, Finland,
France, Netherlands, New Zealand,
Norway, Sweden)
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16. A Pilot Study of MCDA for
Valuing Orphan Medicines
http://www.valueinhealthjournal.com/article/
S1098-3015(13)04356-8/abstract
http://www.ohe.org/publications/article/valuing
-orphan-medicines-using-multi-criteriadecision-analysis-129.cfm
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17. Possible Value Attributes and
Weights (Sussex et al, 2013)
Per cent
‘Experts’
‘Patients’
workshop
workshop
19.5
11
Disease survival prognosis with current soc
14
11.5
Disease morbidity and patient clinical disability with current soc
12
15
Social impact of disease on patients’ and carers’ daily lives with current soc
8
15
53.5
52.5
0
5
27.5
17.5
Treatment safety
8
7.5
Social impact of treatment on patients’ and carers’ daily lives
11
17.5
Sub-total weight for impact of new medicine
46.5
47.5
Total
100
100
Availability of existing treatments
Sub-total weight for impact of disease / extent of unmet need
Treatment innovation: scientific advance + contribution to patient outcome
Evidence of treatment clinical efficacy and patient clinical outcome
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18. Compared with NICE Criteria
Among NICE
criteria?
‘Patients’
workshop
workshop
19.5
11
14
11.5
Disease morbidity and patient clinical disability with current soc
12
15
Social impact of disease on patients’ and carers’ daily lives with current soc
≈ Severity
‘Experts’
Disease survival prognosis with current soc
Standard NO,
HST YES
Per cent
8
15
53.5
52.5
0
5
27.5
17.5
Treatment safety
8
7.5
Social impact of treatment on patients’ and carers’ daily lives
11
17.5
Sub-total weight for impact of new medicine
46.5
47.5
Total
100
100
Availability of existing treatments
{
Sub-total weight for impact of disease / extent of unmet need
No
Treatment innovation: scientific advance + contribution to patient outcome
{
Evidence of treatment clinical efficacy and patient clinical outcome
≈ QALY
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19. Who Listens to “Patient’s Voice”?
(Shah et al, 2013)
•
NICE (England & Wales) –
Yes, sometimes cited in
explanations of
recommendations
•
SMC (Scotland) – No
evidence
•
CED (Ontario) – No evidence
•
HAS (France) – No evidence
•
PBAC (Australia) – No
evidence
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20. Getting in Early – Where Patient
Inputs Can Achieve Most
•
Value greater from patients / carers:
•
•
Aiding better measurement of quality of life
•
Informing better valuation of different qualities of life
(“health states”) and other dimensions of value
•
•
Enabling better understanding of what kinds of quality
of life impacts diseases and treatments have
Changing attitudes to uncertainty
Rather than advocacy / special pleading
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21. For Example…
•
Supporting development of quality of life
instrument for rare diseases (as a group)
•
Supporting research to produce a rare-disease
value framework
•
Inputting to design of clinical trials – what is
measured and how
•
Filling gaps in evidence – by surveying
patients/carers
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22. About OHE
To enquire about additional information and analyses, please contact Jon Sussex at jsussex@ohe.org
To keep up with the latest news and research, subscribe to our blog, OHE News.
Follow us on Twitter @OHENews, LinkedIn and SlideShare.
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©2013 OHE
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