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.
4. Patient-reported Outcome
Measures
â˘
Patientsâ own assessment of their health â purposefully
subjective
â˘
Many well-validated instruments exist that are reliable,
sensitive and widely used
â˘
Simple to complete; quick to analyse
â˘
Repeated âsnap shotsâ of health (e.g. before and after
treatment) can provide a clear picture of changes in health
â˘
Disease specific PROMs: more question items/response
options; focussed on a specific aspect of health
â˘
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
ďą
ďą
ďą
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
ďą
ďą
ďą
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
ďą
ďą
ďą
Pain/Discomfort
I have no pain or discomfort
I have moderate pain or discomfort
I have extreme pain or discomfort
ďą
ďą
ďą
Anxiety/Depression
I am not anxious or depressed
I am moderately anxious or depressed
I am extremely anxious or depressed
Š 2013 EuroQol Group
ďą
ďą
ďą
7. Problems Evaluating
Medicines for Rare Diseases
â˘
Costs not much lower than for other medicines
â˘
Patents last no longer than for other medicines
â but OMP* legislation helps offset that
â˘
Small patient numbers mean low sales volumes
and hence high prices
â˘
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$2011m)
 2,000
 1,500
 1,000
 500
2012
2011
2010
2008
2006
2004
2003
2001
1999
1997
1995
1993
1991
1989
1987
1985
1983
1981
 â
1979
Estimates of mean costs per approved drug (US$m, in 2011prices)
 2,500
Source: Mestre-Ferrandiz et
al. 2012
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10. Accepting Uncertain Evidence
â˘
If payer is risk neutral, and the state should be,
then no problem in principle
â˘
But in practice regulators are risk averse
â˘
And are much more risk averse than sufferers
from severe diseases and their carers
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11. NICEâs Standard Criteria
(weights not revealed)
â˘
Incremental cost effectiveness ratio (ICER) ÂŁ/QALY
â˘
Severity of illness (Rawlins et al, 2010)
â˘
Stakeholder insights (Rawlins et al, 2010; NICE, 2008)
â˘
End of life treatments (Rawlins et al, 2010; NICE, 2008)
â˘
Disadvantaged populations (Rawlins et al, 2010; NICE,
â˘
Children given âbenefit of the doubtâ (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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ÂŁ500,000
ÂŁ100,000
ÂŁ70,000
ÂŁ45,500
ÂŁ50,000
ÂŁ60,000
ÂŁ37,500
ÂŁ40,000
ÂŁ45,000
ÂŁ35,000
ÂŁ32,500
ÂŁ30,000
ÂŁ27,500
ÂŁ20,000
ÂŁ22,500
ÂŁ25,000
ÂŁ17,500
ÂŁ15,000
ÂŁ12,500
ÂŁ10,000
ÂŁ7,500
ÂŁ5,000
ÂŁ2,500
ÂŁ0
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 â 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 â England and Wales
Yes â Interim HST process
(previously AGNSS). Also Cancer
Drugs Fund
SMC â Scotland
No â But Rare Conditions Fund
PBAC - Australia
Life Saving Drugs Program and ârule
of rescueâ though not about rarity
per se
GB-A - Germany
< âŹ1million 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
âExpertsâ
âPatientsâ
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 patientsâ and carersâ 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 patientsâ and carersâ 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?
âPatientsâ
workshop
workshop
19.5
11
14
11.5
Disease morbidity and patient clinical disability with current soc
12
15
Social impact of disease on patientsâ and carersâ daily lives with current soc
â Severity
âExpertsâ
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 patientsâ and carersâ 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
â QALY
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19. Who Listens to âPatientâs Voiceâ?
(Shah et al, 2013)
â˘
NICE (England & Wales) â
Yes, sometimes cited in
explanations of
recommendations
â˘
SMC (Scotland) â No
evidence
â˘
CED (Ontario) â No evidence
â˘
HAS (France) â No evidence
â˘
PBAC (Australia) â No
evidence
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20. Getting in Early â Where Patient
Inputs Can Achieve Most
â˘
Value greater from patients / carers:
â˘
â˘
Aiding better measurement of quality of life
â˘
Informing better valuation of different qualities of life
(âhealth statesâ) and other dimensions of value
â˘
â˘
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 ExampleâŚ
â˘
Supporting development of quality of life
instrument for rare diseases (as a group)
â˘
Supporting research to produce a rare-disease
value framework
â˘
Inputting to design of clinical trials â what is
measured and how
â˘
Filling gaps in evidence â 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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Š2013 OHE
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