Transcript Scottish Medicines Consortium

Scottish Medicines
Consortium Approach to Cancer Medicines
Dr Ken Paterson
BOPA Symposium
13 September 2007
Scottish Medicines
Consortium
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Chairman:
Professor David Webb
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Vice-Chairs: Ms Angela Timoney, Dr
Ken Paterson
Remit
Provide advice to NHS Boards and ADTCs
on comparative and cost-effectiveness of:
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New Medicines
New Formulations of Medicines
Major new indications for Medicines
 80 products (approx) per annum
SMC Membership
Membership (30) - multi-disciplinary,
geographically spread
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Physicians (1° and 2° care)
Pharmacists
Nurse, Economists
Board and Trust Executives, ABPI
Lay & Patient Representatives
NHS QIS
Full declarations of interest
New Drugs Committee
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Membership: Total = 18
Physicians, Pharmacists,
Health Economists
Nurse, Public Health Consultant
 Primarily an Evidence Review Committee
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Chairman: Dr Ken Paterson (Glasgow)
SMC Advice to NHSScotland
3 Categories of advice
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Accepted for use in NHS Scotland
Accepted for restricted use in NHS Scotland
Not recommended for use within NHS
Scotland
Some drugs may also be ‘unique!’
The Aim of Product
Assessments
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Efficacy – does the drug have an effect?
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Effectiveness – does it work in normal
use?
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Cost-effectiveness – how much bang for
the buck!
Submission Content
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Standardised form
Summaries of efficacy/effectiveness and safety
Detailed health economic case
 Cost-utility approach preferred (£ per QALY)
 Budget impact for Scotland (or per 100,000)
 Full explanation of model assumptions
 Linked to Scottish (or UK) data
 Full sensitivity analysis
 Univariate ± probabilistic analyses
Submission data
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All referenced data to be included
 May include unpublished data
 ...including ‘commercial in confidence’
Economic data to be included
Supplementary data – SPC, draft protocols, etc
Clinical Expert Panel
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Important to inform the SMC process
 Impact of disease
 Unmet therapeutic need
 Current therapeutic strategies in Scotland
 Test economic case assumptions
NOT asked “do you want this drug?”
All interests declared
Patient & Public Input
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Patient & Public Involvement Group
Patient group submissions considered at
SMC
 Only ~30% of medicines have a patient
group submission
 Can say things which pharma company
cannot say!
Now actively seeking patient group
submissions
Process timelines
Submission to SMC
SMC Assessment
3/4 Weeks
Report to NDC
last Tuesday/month
10-12 weeks
NDC Report to SMC
first Tuesday/month
SMC Advises NHSScotland
NHS Boards & ADTCs
& Applicant Company
SMC Publishes advice on www
(4 weeks advice to NHSScotland)
www.scottishmedicines.org.uk
4 weeks
2002 - 2007
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382 submissions considered
 2002 – 29
 2003 – 62
 2004 – 74
 2005 – 87
 2006 – 130 (111)
~20% are ‘abbreviated’ subs
Rising proportion of re-submissions
Outcome of Assessments
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Accepted for Use – 34%
Accepted for Restricted Use – 36%
Not Recommended – 30%
No real evidence of change over time
45
40
35
30
% 25
20
Accept
Restrict
No
15
10
5
0
2002
2003
2004
2005
2006
Oncology Assessments
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Fewer RCTs per drug (median 1 v 2)
Longer follow-up (52 wks v 12 wks)
Acceptance rate - 67%
 About half with some restriction, usually to
specialist use
Higher cost per QALY (£15K v £8.5K)
Driven by Budget Impact?
Accept
 Restrict
 No
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£413K
£581K
£743K
(120-760K)
(299-863K)
(366-1100K)
Large overlap suggests budget impact
is not driving SMC decision-making
Obsessed by QALYs?
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Cost per QALY < £20K
 …20% not recommended!
Cost per QALY £20-30K
 …58% not recommended
Cost per QALY plays (appropriately?) a large
role – but not the only consideration!
Special Cancer Issues - 1
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Often scanty phase 3 clinical data
Complex regimens with poly-pharmacy make
comparators hard to define
 RCTs often use comparators different from
current Scottish practice
 May require indirect comparison
Survival benefits often unclear
 Overall v ‘progression-free’ survival
 Extrapolation not clear-cut
Extrapolation Possibilities
End of RCT
Disease free survival
1) Only benefit observed in RCT
Extrapolation Possibilities
End of RCT
Disease free survival
2) ‘Frozen’ at end of RCT
Extrapolation Possibilities
End of RCT
Disease free survival
3) Continuing divergence
Extrapolation Possibilities
Year 5
End of RCT
Disease free survival
4) Limited divergence then ‘frozen’
Extrapolation Possibilities
Year 5
End of RCT
Disease free survival
5) Limited divergence then tapering
Special Cancer Issues - 2
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Quality of life assessment difficult
 Impact of adverse events a problem
 ? revaluation of QoL near life’s end
 ? special benefit with low expectancy
Increased niching by indication
 …more (ultra-)orphan drugs
 …with expectations of “special case”
Rule of Rescue - a rule??
Quality of Life
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Are the impacts of adverse events limited to
when they occur?
With 3 months to live, if you say your QoL is
90%, is that true?
 Are time-trade off/standard gamble useful?
Is 3 months extra life worth more if you’ve
had the diagnosis for 3 months rather than 5
years?
 ? discriminates against certain cancers?
Early Technology Appraisal of
Oncology Drugs
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…is possible
…can be done within similar parameters to
other drugs and technologies
…allows real breakthroughs even at
considerable cost
…does not reward small incremental change
at substantial cost
…can avoid ‘decision blight’ and meet the
timelines of specialists and patients
To consider…
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Is this whole process ethical?
 Would it be ethical NOT to do it?
What should the NHS pay for?
 Life, QoL, feeling better, carer time?
How should specialists react…?
 Is money not spent on drugs to manage
cancer always money wasted?!
What should the threshold £ per QALY be?
How should we handle orphan drugs?
Scottish Medicines Consortium
www.scottishmedicines.org.uk