U.S. Eliminates Special Requirements for Gene Therapy Research

Federal health officials have proposed a policy change that would remove a National Institutes of Health (NIH) oversight panel review from the approval process for gene therapy research applications. Instead, these experiments will be submitted only to the U.S Food and Drug Administration (FDA) for approval, while the NIH oversight board will take on an advisory role.

For decades, gene therapy researchers have looked for ways to treat diseases by removing, adding, or altering patient DNA. The NIH, initially concerned about the unknown risks of gene therapy, established the Recombinant DNA Advisory Committee (RAC) to review research applications for these techniques before submission to the FDA. The new policy eliminates NIH’s role, aligning the review process for gene therapy with that of other drugs and treatments.

The change indicates a recognition that gene therapy is now an established form of medical care without extraordinary risks. Some experts, however, dispute the claim. Bioethicist Mildred Cho, PhD, a member of the RAC, expressed concern that the committee would no longer have special oversight of gene therapy research. “There are miraculous recoveries and remissions that we haven’t seen before, but there also are very spectacular failures,” she said.

Others, including Jeffrey Kahn, PhD, director of the Berman Institute of Bioethics at Johns Hopkins University, think the time to normalize the gene therapy review process has come. “We have mechanisms in place to protect patients,” he said. “It doesn’t need to be treated as a special case of clinical research any longer.”

Source: Associated Press, August 15, 2018.