A new collaboration between the National Institutes of Health (NIH) and the US Food and Drug Administration (FDA) seeks to reduce duplicative oversights in gene therapy submissions by eradicating the need for Recombinant DNA Advisory Committee (RAC) review as well as NIH reporting for gene-therapy protocols.

According to a perspective article published in the New England Journal of Medicine, Francis S. Collins, MD, PhD, and Scott Gottlieb, MD, reported that, as of 2017, a total of 3 gene therapies have been approved for use in the United States: 2 of which are cell-based and 1 of which is an in vivo therapy used to treat RPE65 mutations associated with retinal dystrophy. Currently, more than 700 active investigational gene therapy products have been submitted to the FDA for review and ultimate approval. As gene therapy continues to evolve, the oversight capabilities for these therapies are beginning to change in kind.

During the summer of 2018, new guidance from the FDA was released relating to gene therapy manufacturing, long-term clinical follow-up, and specific strategies for clinical development in indications such as hemophilia, ophthalmology, and rare diseases. Although both the NIH and FDA have persistently worked to avoid overlap in the approval process, duplication has occurred during submissions of protocols, amendments, annual reports, and serious adverse event reports.

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In the recently proposed elimination of the RAC and the need for reporting protocols to the NIH for the purposes of reducing duplication, the FDA and NIH are also seeking to revise institutional Biosafety Committees’ responsibilities so that their review of gene-therapy protocols will reflect that of other research reviewed by the NIH. In doing so, the NIH believes this will create a streamlined process that “will also appropriately place the focus of the NIH Guidelines squarely back on laboratory biosafety.”

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The original goal of the RAC “was to advise the NIH director on the scientific, safety, and ethical issues associated with emerging biotechnology,” Drs Collins and Gottlieb wrote. “With the continued emergence of new biotechnologies beyond the realm of recombinant DNA, the RAC’s role must evolve.”


Collins FS, Gottlieb S. The next phase of human gene-therapy oversight [published August 15, 2018]. N Engl J Med. doi: 10.1056/NEJMp1810628