Access to Drugs for Rare Diseases: Canada and International Perspectives

1. Tania Stafinski, MSc, PhDDev Menon, MHSA, PhDDerek Clark, BScN, MPHHilary Short, BSc, MSc TOWARDS APPROPRIATE AND SUSTAINABLE ACCESS TO DRUGS FOR RARE DISEASES (DRDs): WHERE ARE WE NOW IN CANADA AND WHAT CAN WE LEARN FROM ABROAD?

2. OVER THE NEXT 12 MINUTES… • Where are we now in Canada? - Current mechanisms for determining which DRDs to provide - Impact on access - Current challenges - Efforts towards a pan-Canadian approach • What can we learn from international experience? - Similarities and differences in healthcare systems - Current challenges • Where could we go from here?

3. WHERE ARE WE NOW IN CANADA? DRD manufacturer application for reimbursement CADTH pCODR/CDR Pan-Canadian Pharmaceutical Alliance (pCPA) Drug plans within participating jurisdictions

4. WHERE ARE WE NOW IN CANADA? Reimbursement decision-making mechanism General reimbursement process Case by case review 1 DRD specific program 1 1Uses Non-Insured Health Benefits Program general reimbursement review process

5. WHERE ARE WE NOW IN CANADA? • DRD specific programs • - 5 provinces • - 2 types Separate plan containing defined set of DRDs Separate decision-making processes for DRDs • Alberta’s Rare Disease Drug Coverage Program • New Brunswick’s Drugs for Rare Diseases Plan • BC’s Expensive Drugs for Rare Diseases (EDRD) Advisory Committee • Ontario’s DRD Evaluation Framework and DRD Working Group • Saskatchewan’s EDRD Process

6. WHERE ARE WE NOW IN CANADA? Proportion of CDR-reviewed DRDs (non-oncology) reimbursed in each province/territory through one of three mechanisms

7. WHERE ARE WE NOW IN CANADA? Degree of agreement on reimbursement status across jurisdictions by Anatomic, Therapeutic and Chemical Classification (ATC) group Kappa score interpretation < 0 Less than chance agreement 0.01–0.20 = Slight agreement 0.21– 0.40 = Fair agreement 0.41–0.60 = Moderate agreement 0.61–0.80 = Substantial agreement 0.81–0.99 = Almost perfect agreement

8. WHERE ARE WE NOW IN CANADA? • Current challenges Many DRDs High per patient cost ? Sustainability ? Value ? Clinical benefit

9. WHERE ARE WE NOW IN CANADA? • Efforts towards appropriate and sustainable access to DRDs • 1. Regulatory level • - Vanessa’s Law • - Draft Orphan Drug Regulatory Framework • 2. Reimbursement level • - Pan-Canadian framework for EDRD • - Pan-Canadian plan for rare diseases • - pCPA • - Improved access to pharmaceuticals one of the priorities of new gov’t • - Aplace in the Health Accord?

10. WHAT CAN WE LEARN FROM INTERNATIONAL EXPERIENCE? Proportion of DRDs with regulatory approval reimbursed by country Source:

11. WHAT CAN WE LEARN FROM INTERNATIONAL EXPERIENCE? National price versus mean EU price in countries with greatest number of reimbursed DRDs Source:

12. WHAT CAN WE LEARN FROM INTERNATIONAL EXPERIENCE? Reimbursement decision-making processes in the same four countries

13. WHERE DO WE GO FROM HERE? • Dealing with uncertainties in clinical benefit • - Registries? • - MEAs – payment by clinical results? • Dealing with high prices • - MEAs – payment by financial results? • - Discounts? • - Single Canadian purchasing – increased buying power?

14. THANK YOU! Contact: Tania StafinskiE-mail: tanias@ualberta.caTel: 780 492 4791