Canada’s Drug Agency Announces Recipients of Funding to Enhance Rare Disease Registries

Canada’s Drug Agency is pleased to announce the successful applicants from our 2024 Request for Proposals (RFP) to support the enhancement of rare disease registries in Canada. The awarded rare disease registries span diverse disease areas and will undertake varied improvement initiatives, all with the goal of generating high quality and fit-for-purpose data that can be used to help answer regulatory and reimbursement questions related to drugs for rare diseases.

Selection Process

In March 2024, Canada’s Drug Agency launched an open call for time-limited funding for registry owners, through an RFP. More than 60 applicants expressed interest in this new funding opportunity, with 36 organizations invited to submit detailed proposals outlining their proposed objectives and workplans, and capability to deliver the intended registry enhancements. An internal panel at Canada’s Drug Agency with expertise and experience in real-world evidence methods, disease registries, and the pan-Canadian decision-making landscape evaluated all proposals, and 18 organizations with the highest scores were awarded funding.

The panel evaluated and ranked the submissions by scoring domains that assessed immediate impact to health care decision-making needs, strategic importance for advancing the evidence landscape for rare diseases, and feasibility to execute the proposed initiatives, among other domains described in the RFP. The 18 registries span several rare disease areas, and the funding allocations range from approximately $100,000 to $200,000.

Information about the successful applicants is now published on our website and includes organization names, lead applicant(s), and funding amounts.

We want to extend our sincere appreciation to all applicants who submitted proposals. Your efforts and innovative ideas made this a highly competitive selection process.

How The Funds Will be Used to Support Improvement Initiatives

In recognition that rare disease registries across Canada are at different levels of development and maturity, the funds will support a diverse range of objectives aimed at enhancing registries. The improvements may include technological infrastructure upgrades, enhancing registries’ policies and key document development, expanding data coverage, and improving capabilities to support decision-grade analyses. Collectively, these initiatives will serve to improve the pan-Canadian evidence landscape and help support the National Strategy for Drugs for Rare Diseases.

Next Steps

This time-limited funding runs until March 31, 2025.

This funding opportunity is one of several initiatives led by Canada’s Drug Agency to improve the collection and use of evidence to inform and support rare disease decision-making. This work is undertaken in support of, and with funding from, the Government of Canada’s National Strategy for Drugs for Rare Diseases.

About Rare Disease Registries

Disease registries are centralized repositories, which collect observational data about a group of patients who share a specific disease or condition and are important sources of information for the evaluation of treatments for rare diseases. The real-world data from these registries can be especially important to inform regulatory and reimbursement decision-making, as rare diseases affect a small patient population, and it can be challenging to recruit participants and conduct traditional clinical trials.