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Ganging up in rare disease

What has to happen to implement EMA/FDA proposal for platform trials in rare disease

A joint proposal from EMA and FDA that encourages companies to join multidrug platform trials for pediatric Gaucher’s disease came too late to serve its original purpose. But the proposal could provide a template for other rare diseases where multiple therapies compete for few patients, if stakeholders can overcome logistical and operational hurdles.

EMA published its final version of the proposal July 3, and FDA plans to publish a draft version of the proposal for public comment “in the next few months,” according to an agency statement.

The proposal was developed over six years with input from Japanese and Canadian regulators, patient advocacy groups, industry and healthcare providers who treat Gaucher’s.

EMA and FDA began the project in 2011 after realizing that the pediatric investigation plans agreed upon for three Gaucher’s treatments newly approved or in Phase III testing at the time could require enrolling over half of the children with Gaucher’s in Europe.

“You hope to get to the finish line first with a meaningfully efficacious product to get exclusivity.”


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