Tania Stafinski, MSc, PhD Dev Menon, MHSA, PhD Derek Clark, BScN, MPH Hilary 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 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

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?

Country Reimbursement 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?

THANK YOU! Contact: Tania Stafinski E-mail: tanias@ualberta.ca Tel: 780 492 4791