Specific, Fair and Transparent: A Canadian Process for Funding Drugs for Rare Diseases

Area of Science:

• Health Economics
• Pharmaceutical Policy
• Rare Disease Research

Background:

• Traditional evaluation methods and value-for-money focus in Canada create barriers for funding drugs for rare diseases (DRDs).
• Existing frameworks may not adequately address the unique needs and evidence requirements for DRDs.

Purpose of the Study:

• To validate and extend recommendations for improving DRD funding in Canada.
• To propose enhancements for evidence generation and funding frameworks for DRDs.

Main Methods:

• Commentary and extension of existing recommendations from a lead paper (Sirrs et al. 2023).
• Incorporation of patient-reported outcomes and data capture for treatment response.
• Argument for specific public and private payer guidance and a dedicated DRD funding structure.

Main Results:

• The necessity of a pan-Canadian approach for collecting robust evidentiary data for DRDs is reinforced.
• The importance of enabling patients to actively participate in tracking and measuring treatment response through data capture is highlighted.
• The need for a distinct, fair, and transparent funding framework for DRDs, with clear public and private payer guidance, is advocated.

Conclusions:

• Implementing a pan-Canadian data collection strategy, including patient-generated data, is crucial for evaluating DRDs.
• A specialized, transparent funding framework is essential to overcome current challenges and ensure equitable access to DRDs in Canada.