The U.S. Food and Drug Administration (FDA) has recently published a number of draft and final Guidance for Industry documents of relevance to developers/manufacturers of cell- and gene-based therapies. These include:
- Analytical Procedures and Methods Validation for Drugs and Biologics. Guidance for Industry (link to document)
- Rare Diseases: Common Issues in Drug Development. Guidance for Industry (link to document)
- Recommendations for Microbial Vectors used for Gene Therapy. Draft Guidance for Industry (link to document)
- E6(R2) Good Clinical Practice. Draft ICH Consensus Guideline (link to document)
- M4E(R2): The CTD – Efficacy. Draft ICH Consensus Guideline (link to document)
In addition, a team of researchers led by statisticians at the FDA has published guidelines on how to present clinical trial safety data in a clear and compelling manner with minimal explanatory text. The guidelines appear in the June 25, 2015 issue of Statistics in Medicine, and further details can be found here.
Author: Anthony Lodge