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The Advisory Committee for Therapeutics (ACT) toolkit provides procedural advice and guidance to replicate the successful TREAT-NMD Advisory Committee for Therapeutics (TACT) model in other rare disease communities. An ACT can provide independent and objective advice to industry and academia, on a real-life therapeutic pathway of drug development programmes. An ACT review can help an applicant to position a candidate compound along a realistic and well-informed pathway to clinical trial and eventual registration. 

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Rare Diseases Clinical Research Network (RDCRN)

Published by NCATS

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The RDCRN program is designed to advance medical research on rare diseases by providing support for clinical studies and facilitating collaboration, study enrollment and data sharing. Through the RDCRN consortia, physician scientists and their multidisciplinary teams work together with patient advocacy groups to study more than 200 rare diseases at sites across the nation.

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Japan Agency for Medical Research and Development (AMED) leads the Initiative on Rare and Undiagnosed Diseases (IRUD) program. The program provides patients across Japan suffering from undiagnosed diseases with a diagnostic strategy that involves genetic analysis

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At the FDA, the Patient Affairs Staff (PAS) is devoted to supporting cross-cutting patient engagement activities across the FDA. Its mission is to coordinate and support patient engagement activities across FDA to facilitate awareness and collaboration with patients, their advocates and the FDA

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