EMA, FDA propose ideas to expedite Gaucher's development

On Monday, EMA and FDA jointly proposed new approaches designed to expedite development of treatments for Gaucher's disease, including use of data extrapolation and clinical trials that test more than one therapy against a single control. The agencies said the approaches could be applied more broadly to development of drugs for other rare pediatric diseases.

The agencies said that under certain conditions, extrapolation of efficacy in pediatric Gaucher's disease from studies in adults or other pediatric populations "can be considered." The proposal said that by using tools such as modeling and simulation, companies could avoid unnecessary studies and reduce testing burden on patients...