c2 stafinski
TRANSCRIPT
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?
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?
WHERE ARE WE NOW IN CANADA?
CADTH pCODR/CDR
Pan-Canadian Pharmaceutical Alliance (pCPA)
Drug plans within participating jurisdictions
DRD manufacturer application for
reimbursement
WHERE ARE WE NOW IN CANADA?
General reimbursement process
Case by case review
DRD specific program
1
1
1Uses Non-Insured Health Benefits Program general reimbursement review process
Reimbursement decision-making mechanism
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
WHERE ARE WE NOW IN CANADA?
Proportion of CDR-reviewed DRDs (non-oncology) reimbursed in each province/territory through one of three mechanisms
WHERE ARE WE NOW IN CANADA?
Degree of agreement on reimbursement status across jurisdictions by Anatomic, Therapeutic and Chemical Classification (ATC) group
ATC Group Kappa
Alimentary tract and metabolism products 0.051
Antineoplastic and immunomodulating agents 0.57
Blood and blood-forming organ agents 0.11
Cardiovascular system agents 0.45
Musculoskeletal system agents 0.49
Nervous system agents 0.71
Respiratory system agents -0.083
Systemic anti-infective agents 0.15
Systemic hormonal preparations 0.17
All DRDs (non-oncology) 0.48
Kappa score interpretation< 0 Less than chance agreement0.01–0.20 = Slight agreement 0.21– 0.40 = Fair agreement 0.41–0.60 = Moderate agreement 0.61–0.80 = Substantial agreement0.81–0.99 = Almost perfect agreement
WHERE ARE WE NOW IN CANADA?
• Current challenges
? Clinical benefit
High per patient cost
Many DRDs
? Value
? Sustainability
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 - A place in the Health Accord?
WHAT CAN WE LEARN FROM INTERNATIONAL EXPERIENCE?
Proportion of DRDs with regulatory approval reimbursed by country
Source:
WHAT CAN WE LEARN FROM INTERNATIONAL EXPERIENCE?
National price versus mean EU price in countries with greatest number of reimbursed DRDs
Source:
WHAT CAN WE LEARN FROM INTERNATIONAL EXPERIENCE?
CountryReimbursement decision-
making mechanisms
DRD specific mechanisms or considerations
Conditions of reimbursement: Conditional listing
agreements/Managed Entry Agreements (MEAs)
France • General reimbursement• Case by case (individual or cohort)
None - Frequently used- Common framework for all MEAs based on payment by results (financial)- Few payment by results (clinical)
Netherlands • General reimbursement No economic evaluation required
- Frequently used- In past, mainly coverage with evidence development - Increasing use of payment by results (clinical)
Denmark • General reimbursement• Case by case (individual or cohort)
None - Rarely used- Not formally a part of reimbursement system
Italy • General reimbursement• Case by case (individual or cohort)
AIFA 5% Fund - Frequently used- Payment by results (mainly clinical)- Monitoring registries
Reimbursement decision-making processes in the same four countries
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?
