FDA, NIH Reducing Regulatory Oversight of Gene Therapy Trials
FDA and NIH are proposing to relax some oversight of gene therapy trials to help expedite product development. Writing in a New England Journal of Medicine online perspective, FDA commissioner Scott Gottlieb and NIH director Francis Collins announce their reevaluation of the U.S. oversight system for such trials. “As the NIH, the FDA, and research entities have moved to strengthen their individual oversight efforts, some overlaps have occurred,” they say. “For example, substantial duplication has arisen in the submission of initial protocols, annual reports, amendments, and reports of serious adverse events. Originally, these overlaps — which affect no other field of biomedical research — were viewed as harmonized reporting that enabled the FDA to conduct regulatory oversight while maintaining confidentiality with sponsors and allowed the NIH to provide transparency with regard to the research. But the intervening implementation of ClinicalTrials.gov has resulted in a high level of transparency for many gene-therapy trials conducted by both public and private sponsors.”
Gottlieb and Collins acknowledge the gene therapy field’s rapid evolution — FDA currently has more than 700 active investigational new drug applications for gene therapies. They also note that in July FDA released a suite of draft guidance documents pertaining to gene therapy that proposes new guidance on manufacturing issues, long-term follow-up, and pathways for clinical development in certain areas, including hemophilia, ophthalmologic indications, and rare diseases.
In changes proposed to be released this week in the Federal Register, NIH and (XXX DELETE XXX)FDA seek to reduce the duplicative oversight burden by further limiting the role of the NIH and its Recombinant DNA Advisory Committee (RAC) in assessing gene-therapy protocols and reviewing their safety information. “Specifically, these proposals will eliminate RAC review and reporting requirements to the NIH for human gene-therapy protocols,” Gottlieb and Collins write. “They will also revise the responsibilities of institutional Biosafety Committees, which have local oversight for this research, making their review of human gene-therapy protocols consistent with review of other research subject to the NIH Guidelines. Such streamlining will also appropriately place the focus of the NIH Guidelines squarely back on laboratory biosafety.”
The agency leaders say there will be an opportunity to return the RAC to the spirit in which it was founded. “Its original goal was to advise the NIH director on the scientific, safety, and ethical issues associated with emerging biotechnology,” they say. “With the continued emergence of new biotechnologies beyond the realm of recombinant DNA, the RAC’s role must evolve. The NIH envisions using the RAC as an advisory board on today’s emerging biotechnologies, such as gene editing, synthetic biology, and neurotechnology, while harnessing the attributes that have long ensured its transparency. We at the NIH and the FDA look forward to working together with all our stakeholders to implement these changes.”