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Executive Summary

NIH's RAC WILL REVIEW SINGLE-PATIENT GENE THERAPY PROTOCOLS ("compassionate use" plea requests) through the Recombinant DNA Advisory Committee's traditional review process unless an emergency health situation warrants expedited action, the committee concluded at a Jan. 14 meeting at the National Institutes of Health. The special meeting of RAC to discuss a mechanism for approving gene therapy protocols for single patients follows NIH's Dec. 28 first-ever approval of a compassionate use exception for gene therapy to treat a San Diego woman suffering from a life-threatening brain tumor. The committee agreed by a nine-to-three vote with one abstention to a skeletal policy statement, devised by committee member Nancy Buc of the D.C. law firm Weil, Gotshal and Manges, that RAC will consider single-patient protocols through its regular review process and will not distinguish between whether the protocol is for research or therapy purposes. In addition, the committee agreed that the criteria required in a protocol submission to RAC should not differ between a single-patient protocol and one that is for a trial consisting of multiple subjects. However, Buc noted that in "emergency situations," where RAC is unable to convene and a patient's condition warrants an immediate decision, "NIH will announce that it will review the protocol itself...applying as best it can the same criteria that the RAC would apply." Buc pointed out that NIH "can illustrate its determination to apply the criteria and favor the RAC, wherever possible if need be, by turning them [the proposed emergency basis single-patient protocols] down." NIH "can just say no. They will have to say no," Buc asserted. When NIH is called upon to review an emergency protocol, the agency is expected to consider the pending protocol's relationship to existing protocols. NIH should consult "to the extent legally and practically possible" with RAC members and NIH experts as well as other outside experts, the policy states. NIH would report back to RAC at its next meeting. Buc indicated that NIH's reporting back to RAC would "allow the best available form of public participation and public review." In addition, it was suggested that investigators report to the committee the status of the research effort. Buc predicted that if "NIH's decisions are a rough proxy for what the RAC would have done will be okay." She added, however, that "if the decision is not a rough proxy... NIH would hear it loud and clear from RAC," and public opinion would "act to reduce the divergence one way or another." The preliminary policy statement will be developed further and presented at RAC's next meeting, which is scheduled for March 1-2. A finalized policy, approved by the NIH director, would then most likely be published in the Federal Register as a proposed amendment to the "NIH Guidelines for Research Involving Recombinant DNA Molecules." The guidelines, which have been in place since 1986, govern recombinant DNA research within the United States. In the meantime, NIH Director Bernadine Healy informed the committee of a short-term, stop-gap approach until a more permanent policy is created to "respond compassionately to imminently dying patients referred by physicians requesting gene therapy." The plan requires: that NIH respond to all compassionate plea requests; that the response be in a timely manner; that NIH gather all appropriate information on patient and public safety through its Office of Recombinant DNA Research; and that FDA and the local institutional review board (IRB) and biosafety committees must also approve the protocol before NIH makes a final decision. RAC member Brigid Leventhal, MD, Johns Hopkins Hospital, expressed concern about the number of exceptions that might be requested through the development of a single-patient protocol mechanism. She stated: "I think many of you are naive in terms of the personality of clinical investigators if you don't think that every single person who wants to do a clinical protocol is not going to show up at the last minute asking for permission." Leventhal warned that RAC's proposed policy "is only adequate in the rare exception." Referring to an earlier suggestion at the meeting that Healy has over 100 compassionate use requests "sitting on her desk," Leventhal urged the committee "to be extremely careful that this mechanism...does not overwhelm [RAC's] routine business." Dusty Miller, PhD, Fred Hutchinson Cancer Research Center, declared that "the original RAC guidelines are entirely suitable. I think we should try to strengthen them, if anything, and make provisions for faster review of protocols under the old guidelines ...I don't have faith in the limited review of NIH or FDA for that matter." FDA's Henry Miller, MD, from the Office of Biotechnology, said the agency would strongly endorse the policy. He said the agency "would clearly favor the adaptability of therapies to an appropriate and relevant time."

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