On December 6, 2017, FDA announced a new approach for pediatric disease drug development that the Agency believes will reduce the number of patients treated with a placebo and lead to more time-efficient product development. According to FDA Center for Drug Evaluation Director Janet Woodcock, M.D.,

FDA is committed to helping develop drugs for rare diseases–especially rare pediatric diseases where unmet needs exist. Working with our European regulatory colleagues at the EMA, FDA has drafted an approach to pediatric rare disease drug development that could eliminate the need for certain clinical studies and, when pediatric clinical studies are needed, could reduce the total number of patients who would receive a placebo instead of a potentially helpful drug. This draft guidance aims to enhance the efficiency of drug development, while minimizing the number of patients required in the trials. Ultimately, we hope to promote new, creative approaches to drug development for Gaucher and other rare pediatric diseases.

FDA’s Guidance, “Pediatric Rare Diseases—A Collaborative Approach for Drug Development Using Gaucher Disease as a Model” describes a new approach for companies to collaborate and test multiple drug products in the same clinical trials. A single control group may be used to compare the safety and efficacy of more than one investigational drug using controlled, multi-arm, multi-company trials. The Guidance also encourages the use of extrapolation of available clinical data through modeling and simulation to predict how a drug may work in children and adolescents based on studies conducted in adults and older pediatric populations.

The Guidance states that for drug development for pediatric rare diseases, it may be necessary to develop, validate, and employ age-specific endpoints, which should be identified as early as possible in a clinical program. FDA further recommends qualification and standardization of clinical outcome assessments, which may be discussed with FDA, relevant stakeholders, and patients. Finally, the Guidance provides a regulatory model, using Gaucher Disease, to explain its thinking for this new approach. FDA is accepting comments on this Guidance until February 5, 2018.