FDA In Brief: FDA Requests Input on Rare Disease Clinical Trial Networks | FDA

FDA In Brief: FDA Requests Input on Rare Disease Clinical Trial Networks | FDA

Today the U.S. Food and Drug Administration (FDA) is opening a docket (FDA-2020-N-0837) to obtain input from the public and stakeholders on the establishment of a rare disease clinical trial network. Specifically, we are interested in learning practical steps and successful approaches relating to start-up, implementation and sustainment of clinical trial networks for rare diseases. To support innovation and quality in the drug development pipeline for rare diseases, FDA is establishing   a “Rare Disease Cures Accelerator.” Through the Rare Disease Cures Accelerator, FDA is looking to provide a more cooperative approach and common platform(s) and approaches to support: 1) rare disease characterization, 2) development of standard core sets of clinical outcome assessments and endpoints relevant to rare conditions, and 3) support conduct of clinical trials in rare disease populations. Following FDA receipt of $10 million in FY 2019 Congressional appropriations for investment and innovation for rare diseases, FDA launched a set of efforts to begin building capabilities for the first two of these three components. To learn more, please visit FDA’s Rare Disease Cures Accelerator Homepage. With this request for information and comments, FDA is interested in understanding what work is currently being done and what work needs to be done to address the third component of its Rare Disease Cures Accelerator - improving the design, conduct and completion of rare disease clinical trials. Because of the small size of rare disease populations and global occurrence of rare conditions, networks needed to support rare disease drug development would also have global reach and operations. FDA looks forward to receiving comments on establishing clinical trials networks from stakeholders via the associated docket (FDA-2020-N-0837). More information on FDA’s efforts pertaining to rare diseases is available on FDA’s Office of Orphan Products Development and Center for Drug Evaluation and Research’s Rare Diseases Program websites.

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