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  1. FDA In Brief

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

May 29, 2020

Media Inquiries

  Amanda Turney

The following quote is attributed to Anand Shah, M.D., FDA Deputy Commissioner for Medical and Scientific Affairs:

“The FDA is unleashing innovation for the development of new and better treatments for rare diseases.

“For the approximately 7,000 known rare diseases, less than 10% have an FDA-approved treatment available. As drug development for rare diseases can be challenging due to the small number of patients and limited understanding of the variability and progression of the diseases, the FDA is committed to developing bold new approaches to harness the infrastructure of global clinical trial networks.

“Today’s announcement is part of our continued work on the Rare Disease Cures Accelerator, which combines scientific expertise with patient perspectives to advance innovation and quality in the drug development pipeline ”

  • Today the U.S. Food and Drug Administration announced a Request for Information and Comment on Rare Disease Clinical Trial Networks. In particular, the FDA is requesting public input on practical steps and successful approaches relating to the start-up, implementation and sustainment of global clinical trials networks, including specific considerations for establishing such networks for a range of rare diseases. Comments will be accepted for 60 days following publication of the docket, FDA-2020-N-0837.
  • Today’s request for comments is part of the FDA’s continued work toward building the Rare Disease Cures Accelerator, a cooperative scientific initiative for the development of common, standardized platforms that can improve the characterization of rare diseases, incorporate the patient’s perspective in clinical outcome assessment measures and build clinical trial readiness in the pre-market space.


The FDA, an agency within the U.S. Department of Health and Human Services, protects the public health by assuring the safety, effectiveness, and security of human and veterinary drugs, vaccines and other biological products for human use, and medical devices. The agency also is responsible for the safety and security of our nation’s food supply, cosmetics, dietary supplements, products that give off electronic radiation, and for regulating tobacco products

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