Pediatric Rare Diseases-A Collaborative Approach for Drug Development Using Gaucher Disease as a Model; Draft Guidance for Industry; Availability

The Food and Drug Administration (FDA or Agency) is announcing the availability of a draft guidance for industry entitled ``Pediatric Rare Diseases--A Collaborative Approach for Drug Development Using Gaucher Disease as a Model.'' This draft guidance focuses on drug development for pediatric patients with Gaucher disease. In particular, it proposes for consideration a novel approach to improve the efficiency of drug development in pediatric rare diseases using Gaucher disease as an example. The emergence of concomitant trials for multiple investigational drug products for the treatment of rare diseases can pose significant challenges to effective drug development, because there are limited numbers of patients for any given rare condition worldwide. This approach discusses the feasibility of the development of multiple drug products in a time-efficient manner while minimizing the number of patients necessary to be treated with placebo.