FDA, Gene Therapy, Regulatory

FDA, NIH look to streamline oversight of gene therapies

The FDA and NIH are proposing to further limit the role of the NIH and its Recombinant DNA Advisory Committee (RAC) in the review of human gene therapies.

The changes, announced in the Federal Register on Thursday, are aimed at reducing overlaps in the two agencies’ oversight of gene therapies which result in duplicative work for sponsors as the field progresses following the approval of the first three gene therapies last year.

Scott Gottlieb

In an accompanying perspective by FDA Commissioner Scott Gottlieb and NIH Director Francis Collins in the New England Journal of Medicine, the two agency heads write that there is no longer a compelling need to subject gene therapies to additional oversight compared to other types of biomedical products.

“In the view of the senior leaders of the FDA and the NIH, there is no longer sufficient evidence to claim that the risks of gene therapy are entirely unique and unpredictable—or that the field still requires special oversight that falls outside our existing framework for ensuring safety,” they write.

Currently, sponsors of clinical trials involving gene therapies are required to submit initial protocols, annual reports, amendments and serious adverse event reports to both agencies.

Francis Collins

Historically, the RAC also played a role in reviewing gene therapy protocols and safety data arising from such studies, though in 2016 the NIH limited the committee’s role to protocols “that raised exceptional issues or concerns” based on a recommendation from the Institute of Medicine. According to the NIH, the RAC has only reviewed three of the 275 gene therapy protocols submitted since that change took effect.

However, under the FDA and NIH’s proposal, the RAC’s role in reviewing gene therapy protocols would be entirely eliminated and sponsors would no longer be required to submit reports to NIH on such protocols.

Additionally, the NIH says it will revise the roles of Institutional Biosafety Committees (IBCs), which are tasked with local oversight of studies involving recombinant or synthetic nucleic acid molecules, to ensure their oversight of human gene therapy protocols is consistent with other research subject to the NIH Guidelines.

According to Gottlieb and Collins, advances in the general framework for clinical trial and medical product oversight and the creation of ClinicalTrials.gov have reduced the need for special oversight for gene therapies.

The two also cite other efforts, such as revisions to the Common Rule and the FDA’s release of draft guidance on manufacturing issues, long-term follow up and clinical development pathways for gene therapies, as reducing the need for special oversight of gene therapy protocols by the NIH.

“Even as our understanding of gene therapy has advanced, so has our general framework for medical product safety. The tools we use to address other areas of science are now well suited to gene therapy,” they write.

Going forward, the NIH says the RAC will continue to function as an advisory board to the NIH on emerging fields such as gene editing, synthetic biology and neurotechnology.


First published here. Regulatory Focus is the flagship online publication of the Regulatory Affairs Professionals Society (RAPS), the largest global organization of and for those involved with the regulation of healthcare and related products, including medical devices, pharmaceuticals, biologics and nutritional products. Email news@raps.org for more information. 


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