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NIH Record

Initiatives To Protect Participants in Gene Therapy Trials

The Food and Drug Administration and NIH recently announced two initiatives to bolster safeguards for people enrolled in clinical studies for gene therapy. The new initiatives — the Gene Therapy Clinical Trial Monitoring Plan and the Gene Transfer Safety Symposia — complement and advance current patient protections.

FDA's clinical trials monitoring plan addresses emerging evidence that the monitoring by study sponsors of several recent gene therapy trials has been less than adequate. To buttress the rigor of the oversight, FDA will require that sponsors of gene therapy trials routinely submit their monitoring plans to the agency.

FDA will review these plans and seek modifications as warranted to improve the quality of monitoring. FDA will also perform surveillance and "for cause" inspections of clinical trials to assess whether the plans are being followed and whether monitoring has been adequate to identify and correct critical problems. The sponsors will also have to address such issues as the experience and training of the monitors and the adequacy of the monitoring in their plans. In addition, NIH and FDA will seek to enhance the conduct of gene therapy trials by convening a conference of investigators at which the appropriate monitoring practices will be discussed by the most experienced professionals in the field.

"Clinical trial monitoring and responsible reporting must be taken seriously by all parties involved in gene therapy trials," said commissioner of food and drugs Dr. Jane Henney. "Our plan will help restore the confidence in the trials' integrity that is essential if gene therapy studies are to be able to fulfill their potential."

In a second new initiative, a series of gene transfer safety symposia, NIH and FDA will enhance patient safety by providing critical forums for the sharing and analysis of medical and scientific data from gene transfer research. The symposia, which are expected to take place about four times a year, will bring together leading experts in gene transfer research and give them an opportunity to publicly discuss medical and scientific data germane to their specialties. The first symposium took place during a meeting of the recombinant DNA advisory committee Mar. 8-10.

To further increase their educational outreach efforts, FDA and NIH also will provide support for professional organizations and academic centers interested in holding safety conferences focused on gene therapy. "The knowledge and understanding gained through these safety symposia and educational outreach efforts will guide the conduct of current trials and enhance the design of future gene transfer trials to maximize patient safety," said NIH acting director Dr. Ruth Kirschstein.

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