Proposed Changes Released for Review of Gene Therapy Research

NIH has published a proposal to amend the NIH Guidelines for Research Involving Recombinant or Synthetic Nucleic Acid Molecules (NIH Guidelines) to streamline oversight for human gene transfer research and reduce duplicative regulatory reporting requirements. NIH will also no longer accept new human gene transfer protocols for the protocol registration process or convene the Recombinant DNA Advisory Committee (RAC) to review individual human gene transfer protocols, effective immediately.

The proposed changes seek to remove reporting requirements to the NIH for human gene-therapy protocols, allowing existing FDA guidelines and requirements to take precedent. The changes would also eliminate RAC review as it currently exists and revise the responsibilities of institutional biosafety committees (IBCs), which have local oversight for this research, making IBC review of human gene therapy protocols consistent with review of other research subject to the NIH Guidelines. The changes also implicitly repeal the requirement that the IRB and the IBC separately make determinations whether a protocol needs to have RAC review. Advarra will immediately implement a change in the review process in line with this revision.

In a separate published statement, NIH Director Francis Collins elaborated that, “[d]uring the comment period and effective immediately, the NIH will no longer accept new human gene transfer protocols for the protocol registration process under [NIH Guidelines] or convene the RAC to review individual human gene transfer protocols. The NIH Office of Science Policy will also not accept annual reports, safety reports, amendments or other documentation for any previously registered human gene transfer protocols under the NIH Guidelines. The roles and responsibilities of [local IBCs] will continue as described in the NIH Guidelines. These studies remain subject to FDA and other clinical trial regulations, and only after FDA, IBC, and other relevant approvals are in place can these protocols proceed.”

In commentary published in the New England Journal of Medicine, NIH Director Francis Collins and FDA Director Scott Gottlieb stated, “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.”

Advarra is reviewing IBC policies and processes in light of these changes and is prepared to comply with the immediate changes outlined above. Clients will be notified of additional relevant changes related to any final revisions to the NIH Guidelines as they become available.

For more information, read the complete proposal in the Federal Register. Contact Advarra’s IBC Team with any specific questions.

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