To Give Rare Disease Patients Hope, the FDA Must Re-Focus its Efforts

MedCity News July 25, 2024
Terry Wilcox

To bring cross-agency clinical leaders together, the FDA should establish a Center of Excellence for Rare Diseases. This would bring together specialized staff and resources from multiple review divisions to leverage their expertise about different bodily systems, trial designs, pharmacology, and other specialized fields.

With over 7,000 rare diseases affecting more than 30 million people in the United States, the tight-knit community of rare disease patients, providers, researchers, and peer groups is incredibly supportive, but the regulatory environment has been much less so. It has long been divided by disease-specific research and treatment silos, and most clinical research is disease-specific without overlap or input from other experts in related fields. This fragmented lack of knowledge-sharing across varying rare diseases often...

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