Healthcare Policy

Healthcare Policy 19(4) June 2024 .doi:10.12927/hcpol.2024.27353

Commentary: Which Principles for a National Strategy for Rare Diseases?

Marc-André Gagnon


Lexchin and Sirrs proposed 5 relevant principles to guide the use of federal funding for expensive drugs for rare diseases (EDRDs), including funding of outcomes-based risk sharing agreements (OBRSAs) and pro-active commitment and participation in the generation of high-quality evidence in a transparent way. This rejoinder, however, questions if the federal funding should be used only to buy new drugs or if it could be used as well to develop new drugs. It also examines what OBRSAa would require in terms of institutional capacities to allow the collection of real-world evidence.




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