BioCentury
ARTICLE | Regulation

Ganging up in rare disease

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

July 28, 2017 7:22 PM UTC

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...