Zdrowie i gospodarka · 07/12/2017

FDA Releases Draft Guidance for Industry on Pediatric Rare Diseases Drug Development Using Gaucher Disease as a Model

The Food and Drug Administration (FDA) has released a draft guidance for industry on developing drugs for pediatric patients with Gaucher disease, proposing a novel approach to improve efficiency in drug development for rare diseases. The guidance aims to minimize the number of patients needed to be treated with placebo while developing multiple drug products.