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# Guide on Pediatric Rare Disease Drug Development
- URL: https://www.fdaweb.com/guide-on-pediatric-rare-disease-drug-development/
- Published: 2017-12-07T12:00:00.000Z
- Updated: 2026-09-14T23:06:19.000Z
- Author: David McFarland
- Tags: FDA Policy/General, #legacy-id-D5140286

*Federal Register* notice: FDA has made available a draft guidance entitled “Pediatric Rare Diseases — A Collaborative Approach for Drug Development Using Gaucher Disease as a Model.” The document proposes for consideration a novel approach to improve drug development efficiency in pediatric rare diseases using Gaucher disease as an example. It addresses the challenges that arise when concomitant trials for multiple investigational drug products for treating rare diseases limit the numbers of patients for any given rare condition worldwide. To view the notice, click [here](https://www.gpo.gov/fdsys/pkg/FR-2017-12-07/pdf/2017-26357.pdf?ref=fdaweb.com).