BRIEF—FDA draft guidance proposes collaborative approach to help develop drugs for rare pediatric diseases

7 December 2017

The US Food and Drug Administration yesterday issued draft guidance outlining a potential novel approach to develop new drug therapies for rare pediatric diseases.

While Gaucher disease is the focus of the draft guidance, the purpose of the guidance is to facilitate drug development for rare pediatric diseases in general and promote the exploration of efficient drug development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.

The proposed approach discusses the following:

modeling and simulation to optimize the design and inclusion of pediatric studies and inform dosing rationales;
proposed extrapolation from studies in adults to demonstrate efficacy and reduce the testing and evaluation burden for children; and,
the possibility of using a single control group in a multi-arm, multi-sponsor clinical trial as the basis for comparing the safety and effectiveness of more than one investigational drug.

The draft guidance is based on a proposal developed jointly by the European Medicines Agency (EMA) and the FDA.

The FDA is accepting comment on the draft guidance, “ Pediatric Rare Diseases – A Collaborative Approach for Drug Development Using Gaucher Disease,” until February 5, 2018. The draft guidance is available on the FDA’s website.