Scottish Medicines Consortium - Approach to Cancer Medicines

1. Scottish Medicines Consortium -Approach to Cancer Medicines Dr Ken Paterson BOPA Symposium 13 September 2007

2. Scottish Medicines Consortium • Chairman: Professor David Webb • Vice-Chairs: Ms Angela Timoney, Dr Ken Paterson

3. Remit Provide advice to NHS Boards and ADTCs on comparative and cost-effectiveness of: • New Medicines • New Formulations of Medicines • Major new indications for Medicines • 80 products (approx) per annum

4. SMC Membership Membership (30) - multi-disciplinary, geographically spread • Physicians (1° and 2° care) • Pharmacists • Nurse, Economists • Board and Trust Executives, ABPI • Lay & Patient Representatives • NHS QIS • Full declarations of interest

5. New Drugs Committee • Membership: Total = 18 Physicians, Pharmacists, Health Economists Nurse, Public Health Consultant • Primarily an Evidence Review Committee • Chairman: Dr Ken Paterson (Glasgow)

6. SMC Advice to NHSScotland 3 Categories of advice • 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!’

7. The Aim of Product Assessments • Efficacy – does the drug have an effect? • Effectiveness – does it work in normal use? • Cost-effectiveness – how much bang for the buck!

8. Submission Content • 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

9. Submission data • All referenced data to be included • May include unpublished data • ...including ‘commercial in confidence’ • Economic data to be included • Supplementary data – SPC, draft protocols, etc

10. Clinical Expert Panel • 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

11. Patient & Public Input • 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

12. Process timelines 10-12 weeks 4 weeks

13. 2002 - 2007 • 382 submissions considered • 2002 – 29 • 2003 – 62 • 2004 – 74 • 2005 – 87 • 2006 – 130 (111) • ~20% are ‘abbreviated’ subs • Rising proportion of re-submissions

14. Outcome of Assessments • Accepted for Use – 34% • Accepted for Restricted Use – 36% • Not Recommended – 30% • No real evidence of change over time %

15. Oncology Assessments • 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)

16. Driven by Budget Impact? • Accept £413K (120-760K) • Restrict £581K (299-863K) • No £743K (366-1100K) • Large overlap suggests budget impact is not driving SMC decision-making

17. Obsessed by QALYs? • Cost per QALY < £20K • …20% not recommended! • Cost per QALY £20-30K • …58% not recommended • Cost per QALY plays (appropriately?) alarge role – but not the only consideration!

18. Special Cancer Issues - 1 • 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

19. Extrapolation Possibilities 1) Only benefit observed in RCT Disease free survival End of RCT

20. Extrapolation Possibilities 2) ‘Frozen’ at end of RCT Disease free survival End of RCT

21. Extrapolation Possibilities 3) Continuing divergence Disease free survival End of RCT

22. Extrapolation Possibilities 4) Limited divergence then ‘frozen’ Disease free survival Year 5 End of RCT

23. Extrapolation Possibilities 5) Limited divergence then tapering Disease free survival Year 5 End of RCT

24. Special Cancer Issues - 2 • 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??

25. Quality of Life • 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?

26. Early Technology Appraisal of Oncology Drugs • …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

27. To consider… • 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?

28. Scottish Medicines Consortiumwww.scottishmedicines.org.uk