FDA Releases Three Biotherapies Guidance Documents

The Food and Drug Administration issued three guidance documents Wednesday addressing the development, approval and safety of regenerative medicines and cell and gene therapies. 

Innovative Designs for Clinical Trials of CGT Products in Small Populations

This draft guidance advises sponsors planning clinical trials for cell and gene therapy (CGT) products that target rare diseases or conditions. It provides recommendations for the planning, design, conduct and analysis of CGT trials to facilitate FDA’s assessment of product effectiveness.  

Expedited Programs for Regenerative Medicine Therapies for Serious Conditions

This draft guidance outlines how sponsors can expedite development and review of regenerative medicine therapies for serious or life-threatening conditions. The guidance covers regenerative medicine advanced therapy (RMAT) designation, accelerated approval pathways and opportunities to engage with the Center for Biologics Evaluation and Research.

Postapproval Methods to Capture Safety and Efficacy Data for CGT Products

This draft guidance recommends approaches for collecting postapproval safety and efficacy data for CGT products. It does not address data collected for the purpose of expanding clinical indications.

AABB and its biotherapies committees and subsections will conduct a thorough review of the draft guidances in consideration of comments. AABB encourages members to submit comments for consideration to regulatory@aabb.org.