TOWARDS APPROPRIATE AND SUSTAINABLE ACCESS TO DRUGS FOR RARE DISEASES (DRDs): WHERE ARE WE NOW IN CANADA AND WHAT CAN WE LEARN FROM ABROAD? Tania Stafinski, MSc, PhD Dev Menon, MHSA, PhD Derek Clark, BScN, MPH Hilary Short, BSc, MSc
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? DRD manufacturer application for reimbursement CADTH pcodr/cdr Pan-Canadian Pharmaceutical Alliance (pcpa) Drug plans within participating jurisdictions
WHERE ARE WE NOW IN CANADA? Reimbursement decisionmaking mechanism General reimbursement process 1 Case by case review 1 DRD specific program 1 Uses Non-Insured Health Benefits Program general reimbursement review process
WHERE ARE WE NOW IN CANADA? DRD specific programs - 5 provinces - 2 types Separate plan containing defined set of DRDs Separate decisionmaking 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 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 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
WHERE ARE WE NOW IN CANADA? Current challenges Many DRDs High per patient cost? Clinical benefit? 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? Country Reimbursement decision-making processes in the same four countries Reimbursement decisionmaking mechanisms France General reimbursement Case by case (individual or cohort) DRD specific mechanisms or considerations None Netherlands General reimbursement No economic evaluation required Denmark General reimbursement Case by case (individual or cohort) Italy General reimbursement Case by case (individual or cohort) None AIFA 5% Fund Conditions of reimbursement: Conditional listing agreements/managed Entry Agreements (MEAs) - Frequently used - Common framework for all MEAs based on payment by results (financial) - Few payment by results (clinical) - Frequently used - In past, mainly coverage with evidence development - Increasing use of payment by results (clinical) - Rarely used - Not formally a part of reimbursement system - Frequently used - Payment by results (mainly clinical) - Monitoring registries
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?
THANK YOU! Contact: Tania Stafinski E-mail: tanias@ualberta.ca Tel: 780 492 4791