1. New Drug Approval on Prince Edward Island Iain Smith and Amanda Burke CADTH Symposium, Ottawa, ON 2016-04-12

2. Disclosure  We have no actual or potential conflict of interest in relation to this topic or presentation. 2

3. Objectives  Background  Identifying potential drugs  Creating two “short lists”  Process for combining and ranking  Description of Delphi Technique  Future Improvements o n w a r d 3

4. PEI Demographics  146,000 Islanders  15% population > 65 in 2010*  > 25% 2031 *https://www.cihi.ca/en/gsearch/aging%2Bpopulation 4

5. Health PEI  Responsible for the operation and delivery of publicly funded health services in PEI  Created in July 2010  “One Island Health System” 5

6. Health PEI Public Drug Funding Hospitals  2 main referral hospitals  4 community hospitals  1 inpatient psychiatric facility  Provincial Cancer Treatment Centre PEI Pharmacare  29 drug programs; currently under review

7. Health PEI  Previous formulary decision-making  PEI Pharmacare  Hospitals 7

8. Provincial Drugs and Therapeutics Committee  Established in 2012  Streamlining of formulary approval processes  Partnership with government  Formulary alignment

9. Committee Composition Expanding participation in decision making  Public representation  Multi-disciplinary  Finance  Administration

10. National Review Process for New Drugs  Steps prior to provincial decisions  Health Canada’s approval of a drug ≠ provincial/territorial funding  (+) recommendation from expert advisory committee ≠ funding 10

11. PEI Status  Substantial number of drugs or indications with (+) CDR or pCODR /iJODR recommendation still to be considered for PEI  Master list of all drug submissions through the CDR/pCODR process  Tracks PEI status of expert advisory committee recommendations  Updated monthly 11

12. Starting Point  Omit drugs with (-) recommendations or that have not yet been vetted thru pCPA process  Consider whether (-) recommendation was due to clinical reasons or cost  Separate approval process for drugs that are budget neutral  Funding status of each drug in other provinces is identified 12

13. Starting Point  Focus on drugs that are covered in 5 or more provinces  Recommendations of Atlantic Common Drug Review (ACDR) are also considered  Two short lists  Oncology Drugs  Non-oncology Drugs 13

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15. Combining the Lists  Both lists are provided to voting members of PD&T Oncology and Formulary Review Subcommittees  Relevant links to CDR/pCODR reviews are included where applicable  Participants are asked to rank the drugs via an on-line questionnaire and provide comments/rationale 15

16. Consensus Building  Delphi Technique  Written communication between groups with relevant information/perspectives  No face to face interaction  Reveals issues for greater discussion in the course of this type of decision-making  Responses/perspectives are collected, summarized and shared with group members  Members then make another decision based upon the new information  Adds validity to a very difficult process 16

17. Combining the Lists  Pooled results of round 1 & comments are shared with subcommittee members  Participants are asked to consider the results & re-rank the drugs  Pooled ranking is used to create a final list 17

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19. Limitations  Appears to lack objectivity  Not easily described to the average person  Perception of what constitutes an “expert” at the local level  “I’m not an expert, so I my opinion isn’t relevant”  Some “experts” feel we lack sufficient experts  Many are uncomfortable …. 19

20. “All important decisions must be made on the basis of insufficient data”

21. Challenges  Process works best when participants share rationale/perspectives  Providing the appropriate information to participants is important and remains a challenge  Expanding number of participants  More perspectives, better decisions 21

22. Combining Scientific and Colloquial Evidence for Context-Sensitive Guidance Professional Experience and Expertise Political Judgment Values Resources Pragmatics and Contingencies Lobbyists and Special Interest Groups Habits and Tradition Scientific Evidence Source: Lomas et al, 2005 (Davies 2005)

23. Wisdom of the Crowd CriteriaDescription Diversity of opinion Each person should have private information even if it's just an eccentric interpretation of the known facts. IndependencePeople's opinions aren't determined by the opinions of those around them. DecentralizationPeople are able to specialize and draw on local knowledge. AggregationSome mechanism exists for turning private judgments into a collective decision. - Surowiecki, James (2005). The Wisdom of Crowds.

24. Challenges  Engagement of non-clinical members of committees  Consistent approach to estimating costs  …or value  A means of comparison across treatments is needed 24

25. Future  Adoption of Multi-criteria Decision Analysis  Agreed upon criteria  EVIDEM Collaboration  Criteria are “weighted” (facilitated by Delphi)  Weighted criteria are scored against each therapy under consideration  Results are totaled and ranking is established 25

26. EVIDEM Criteria  Disease severity  Unmet needs  Comparative safety/tolerability  Type of preventive and/or therapeutic benefit  Comparative cost consequences –cost of intervention –other medical costs –non-medical costs  Size of affected population  Comparative effectiveness  Comparative patient- perceived health / PRO  Quality of evidence  Expert consensus/clinical practice guidelines? 26

27. ….to conclude  On PEI we have a somewhat unique situation,  …but also challenges common to many other groups  A process that’s not perfect, but an improvement  A sense that we can do better, and hopefully learn to live within our means