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# FDA Issues Rare Pediatric Disease Guidance
- URL: https://www.fdaweb.com/fda-issues-rare-pediatric-disease-guidance/
- Published: 2017-12-06T12:00:00.000Z
- Updated: 2026-09-14T23:06:03.000Z
- Author: David McFarland
- Tags: Drugs, #legacy-id-D5140275

FDA has issued a draft guidance, *Pediatric Rare Diseases—A Collaborative Approach for Drug Development Using Gaucher Disease as a Model*, to facilitate drug development in pediatric rare diseases. “In particular,” the [document](http://www.fda.gov/downloads/drugs/guidancecomplianceregulatoryinformation/guidances/ucm587660.pdf?ref=fdaweb.com) says, “it discusses a new possible approach to enhance the efficiency of drug development in pediatric rare diseases using Gaucher disease as an example. This new approach consists of a controlled, multi-arm, multi-company clinical trial, which aims to facilitate the development of multiple drug products in a time-efficient manner while minimizing the number of patients necessary to be treated with placebo.”

FDA says the general principles in the guidance should be viewed as a proposal only, and the principles underlying the proposal may be extended to other areas of drug development in rare diseases.

Modified approaches may be proposed, the guidance says, but sponsors should justify the specific choice of each new strategy. “Given that rare disease drug development tends to require global involvement, and the potential differences in requirement between FDA and other regulatory agencies, sponsors are advised to consult the appropriate regulatory agency(ies) prior to initiation of such trials.”