Commentary: Which Principles Should Apply for a National Strategy on Rare Diseases?

Abstract

Lexchin and Sirrs (2024) proposed five relevant principles to guide the use of federal funding for expensive drugs for rare diseases, including funding of outcomes-based risk-sharing agreements (OBRSAs) and proactive commitment and participation in the generation of high-quality evidence in a transparent way. This rejoinder, however, questions whether the federal funding should be used only to buy new drugs or whether it could be used to develop new drugs as well. It also examines what OBRSAs would require in terms of institutional capacities to allow the collection of real-world evidence.