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# Rare Disease Drug Development Guide
- URL: https://www.fdaweb.com/rare-disease-drug-development-guide/
- Published: 2019-02-01T12:00:00.000Z
- Updated: 2026-09-15T00:59:49.000Z
- Author: David McFarland
- Tags: FDA Policy/General, #legacy-id-D5143278

*Federal Register* notice: FDA has issued a draft guidance, *Rare Diseases: Common Issues in Drug Development*, to assist drug and biological sponsors in conducting more efficient and successful drug development programs. The [document](https://www.fda.gov/downloads/Drugs/GuidanceComplianceRegulatoryInformation/Guidances/UCM629579.pdf?ref=fdaweb.com) revises and replaces an 8/2015 drug guidance by updating the natural history studies section; inclusion of issues for evaluation and validation of surrogate biomarkers; description of nonclinical flexibility; additional information on external controls and early randomization; addition of a safety section; retitled chemistry, manufacturing, and controls section to pharmaceutical quality considerations; additional information on changes to drug substance or manufacturing process with clarification on areas of flexibility; and inclusion of an additional considerations section addressing several topics — participation of patients, caretakers, and advocates, consideration of pediatric issues, and interactions with FDA. To view the notice, click [here](https://www.govinfo.gov/content/pkg/FR-2019-02-01/pdf/2019-00677.pdf?ref=fdaweb.com).