Writing Effective Clinical Regulatory Documents For Gene Therapy Trials

By Robin Johnson and Molly Shea, inSeption Group

While each gene therapy program presents unique opportunities and obstacles, the challenges of writing clinical regulatory documents for gene therapy programs can generally be categorized into three key areas: clinical trial design, safety reporting, and template requirements for Biologics License Applications (BLAs) and Marketing Authorization Applications (MAAs).

Trial design contends with small patient populations, inapplicability of a randomized controlled trial structure, and difficult decisions around comparator data. Safety reporting is complicated by the combination of heavy treatment and disease burdens, as well as the challenge of connecting causality dots for regulators. Template requirements often are an exercise in determining the appropriate round holes in each document to insert square pegs of data.