1. NATIONAL INITIATIVES: BEST PRACTICE AND LESSONS LEARNED FROM SCOTTISH EXPERIENCE Alan MacDonald Vice Chairman Scottish Medicines Consortium Hard Choices in Nordic Health Care, Stockholm, November 2015

2. Role of SMC within NHS Scotland Assessment of high cost medicines: End of life and rare conditions Recent changes in process: drivers for change Effects of new processes re acceptance of high cost drugs Reflections on Scottish experience SMC

3. Approx 32 members Doctors (primary/secondary care) Pharmacists Economists CEOs/DoFs Industry Public partners (SMC staff; non voting) SMC: WHO WE ARE

4. Scottish Medicines Consortium SMC: WHO WE ARE

5. Scottish Medicines Consortium SMC: WHAT WE DO New medicines assessment (since 2002) - all new medicines, indications / licence extensions and new formulations (~80 submissions annually) Horizon Scanning (since 2006) -support health board financial planning by providing intelligence on emerging new medicines Scottish Antimicrobial Prescribing Group (SAPG) -national framework to improve the quality of antimicrobial prescribing (“stewardship”)

6. Scottish Medicines Consortium SMC: WHAT WE DO

8. Scottish Medicines Consortium SMC: CONTEXT FOR CHANGE

9. Ivacaftor Targets G551D mutation in CFTR gene Significant improvements in FEV1 v placebo at 24 and 48 weeks Absence of long term data and uncertainty re magnitude of survival ICER approx £330 000 (SEK 4.4million) SMC: MEDICINES FOR RARE CONDITIONS

10. I am proud that Scotland is among the fastest and most efficient medicine review processes anywhere in the world. However, we can’t overlook the concerns raised by clinicians, charities and patients about access to medicines. That is why I commissioned this independent review, which provides some key recommendations on how to improve access arrangements for new medicines in Scotland to make them better than ever before. SMC: MEDICINES REVIEW

11. Process Changes Meeting in Public Company reps present Additional flexibility to improve access to end of life medicines and medicines for rare conditions (PACE, ultra orphan framework) SMC: MEDICINES REVIEW

12. Definitions End of Life Medicine: A medicine used to treat a condition that usually leads to death with 3 years with currently available treatments Orphan medicine: A medicine with EMA designated orphan status (ie conditions affecting fewer than 2 500 people in a population of 5 million) or a medicine to treat an equivalent size of population irrespective of whether it has designated orphan status Ultra orphan medicine: A medicine used to treat a condition with a prevalence of 1: 50 000 or less (or around 100 people in Scotland) SMC: MEDICINES REVIEW

13. QALY Weighting (rejected) Physician and Clinician Engagement (PACE) Option for additional Patient Access Scheme (PAS) at later stage of process

14. Ultra Orphan Framework Nature of the Condition Impact of New Technology Value for Money Patient and Clinician Engagement Impact Beyond Direct Benefits and on Specialist Services Cost to NHS and Personal Social Services SMC: MEDICINES REVIEW

15. PACE Process “The aim of the PACE group is to describe the added benefits of the medicine, from both patient and clinician perspectives, that may not be fully captured within the conventional clinical and economic consideration” SMC: MEDICINES REVIEW

16. Scottish Medicines Consortium SMC: IMPACT OF NEW PROCESSES ACCEPTANCE RATES CategoryAcceptedNot recommendedTotal (n) All submissions775 (73%)289 (27%)1064 Full submissions523 (78%)144 (22%)667 Cancer/Orphan15 (48%)16 (52%)31 Since Introduction of PACE process: Full Submissions71 (79%)14 (21%)85 PACE Medicines25 (69%)11 (31%)36 Non PACE47 (96%)2 (4%)49 Orphan12 (67%)6 (33%)18 Ultra Orphan8 (73%)3 (27%)11

17. Scottish Medicines Consortium SMC: IMPACT OF NEW PROCESSES MEAN COST/QALY * SKEWED BY OUTLIER ** SKEWED BY OUTLIER AcceptedNot recommended Full submissions£32 129£56 362 PACE£35 744£139 232* Non PACE£16 557£18 931 Orphan£32 292£73 691 Ultra Orphan£36 505£324 082**

18. Not Recommended Medicines Trastuzumab/Emtansine Pertuzumab Bevacizumab Abiraterone Cabozantinib (ultra) Olaparib (ultra) Vinflunine Enzalutamide Eribulin Elosulfase Everolimus PS Some drugs may have been further considered after resubmission etc SMC: IMPACT OF NEW PROCESSES

19. Feedback on PACE from Patient Groups “Breakthrough fully supports PACE and believes it has the potential to be the catalyst required by the Scottish Government to increase the number of medicines approved…it is encouraging the SMC is open to listening to the views of stakeholders… (and) has added more flexibility to their processes to give drugs the best change of being approved.” James Jopling, Breakthrough Breast cancer Comment piece, Herald, 14.10.14 SMC: IMPACT OF NEW PROCESSES

20. Challenges to Decision Making Quality Political Context: how do we ensure quality of process in context of “desired” outcome? Meeting in Public: more or less transparency Increased “flexibility”: can we/should we have more structure to the weighting of subjective criteria? SMC

21. Challenges Excessive workload Expectations Ensuring Consistency Engagement Elosulfase (advice issued 7/9/15, ICER approx £800 000!) SMC

22. Recent changes to SMC processes have succeeded in increasing access to medicines for rare diseases and for end of life conditions In addition, new processes have further strengthened the role of patients and public in decision making and have improved transparency Uncertainties remain as to equity of access, whether value for money is being achieved and the particular challenges of very rare conditions SMC: CONCLUSION

23. Thank You for the Invitation SMC

24. Scottish Medicines Consortium OUR FAMILY