FDA, NIH Increase Oversight of Gene Therapy Trials

ROCKVILLE, Md—Stung by the failure of several researchers to fully comply with federal gene therapy rules and reporting procedures, the Food and Drug Administration (FDA) and the National Institutes of Health (NIH) have taken a series of steps to tighten the control and monitoring of such trials.

Most recently, the FDA announced that gene therapy researchers must submit their clinical trial monitoring plans to the agency. FDA said it will review the plans and seek changes to improve a program’s quality where needed. FDA will also conduct inspections of clinical trials to assess whether researchers are following the monitoring plans and whether monitoring has been adequate to identify and correct critical problems.

FDA and NIH will also sponsor symposia about four times a year at which gene therapy researchers will publicly discuss medical and scientific data germane to their specialties and issues important to the success and safety of clinical trials of gene therapy agents.