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The National Institutes of Health (NIH) established the Recombinant DNA Advisory Committee (RAC) in 1974 in response to public concerns about the safety of manipulating genetic material through recombinant DNA. The accumulation of 40 years of experience with gene transfer research has led to a better understanding of the risks. Yet, as gene transfer research has matured, the complexity of the overall regulatory environment has remained. Gene transfer research continues to be subjected to multiple layers of review: the Food and Drug Administration (FDA), institutional review boards, institutional biosafety committees, and the RAC. It is within the context of overlapping regulatory authority and improved scientific understanding and social acceptance that the NIH commissioned the Institute of Medicine (IOM) to assess whether gene transfer research continues to warrant additional oversight.

The overarching goal of the IOM was to ensure patient safety and the ethical conduct of research, while not subjecting scientists to unnecessary regulatory burdens, which can impede or delay scientific exploration and medical innovation. The IOM Committee recommended that the RAC should review individual protocols only if other regulatory authorities could not adequately do so, and the study meets one of the following criteria: (1) the protocol uses a new vector, genetic material, or delivery methodology representing a first-in-human experience; (2) the protocol relies on preclinical safety data obtained using a new preclinical model system of unknown and unconfirmed value; or (3) the protocol involves a vector, gene construct, or method of delivery associated with possible toxicities that are not widely known.

Gene transfer research no longer stands alone as the only human application of an emerging technology that could benefit from additional oversight. Consequently, the IOM Committee recommended that the NIH Director charge a standing or new committee to examine the need for additional or different oversight for clinical applications of emerging novel technologies.

Publication Citation

Lawrence O. Gostin, Future Oversight of Recombinant DNA Research: Recommendations of an Institute of Medicine Committee, JAMA Online (February 19, 2014), 311 JAMA 671-672 (2014)