“FDA draft guidance proposes collaborative approach to facilitate development of drugs for rare pediatric diseases… On December 6, 2017, the FDA issued draft guidance outlining a potential novel approach to develop new drug therapies for rare pediatric diseases. While Gaucher disease is the focus of the draft guidance, the purpose of the guidance is to facilitate drug development for rare pediatric diseases in general and promote the exploration of efficient drug development approaches for Gaucher and other similar rare diseases. FDA is accepting comment on the draft guidance, Pediatric Rare Diseases – A Collaborative Approach for Drug Development Using Gaucher Disease as a Model, until February 5, 2018.” Here’s a link to the draft guidance.
As pharmaceutical consultants engaged in the compilation and submission of marketing applications, we are frequently called upon by our clients to address all or part of their BPCA and PREA needs. This paper is meant to serve as a review and primer for anyone at the Pre-IND stage or who is confronted with the need for help with an iPSP, including a waiver or deferral request.