> ## Content Index
> Fetch the complete content index at: https://www.fdaweb.com/llms.txt
> Use this file to discover other available public pages before exploring further.

# Draft Guide on Rare Disease Gene Therapies
- URL: https://www.fdaweb.com/draft-guide-on-rare-disease-gene-therapies/
- Published: 2018-07-12T12:00:00.000Z
- Updated: 2026-09-15T00:19:07.000Z
- Author: David McFarland
- Tags: FDA Policy/General, #legacy-id-D5141852

*Federal Register* notice: FDA has made available a draft document entitled “Human Gene Therapy for Rare Diseases; Draft Guidance for Industry.” The document provides recommendations on designing clinical development programs for such products where there may be limited study population size and potential feasibility and safety issues, as well as issues relating to the interpretability of bioactivity/efficacy outcomes that may be unique to rare diseases or to the nature of the gene therapy itself. To view the notice, click [here](https://www.gpo.gov/fdsys/pkg/FR-2018-07-12/pdf/2018-14871.pdf?ref=fdaweb.com).