New Initiatives to Protect Participants in Gene Therapy Trials

FOR IMMEDIATE RELEASE Tuesday, March 7, 2000 Contact: FDA Press Office (301) 827-6250 NIH Press Office (301) 496-5787

New Initiatives to Protect Participants in Gene Therapy Trials

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 FDA.

FDA will review these monitoring 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 is a powerful tool in enhancing the safety and protection of research subjects during a trial. Monitors are selected by and report to the sponsor or the sponsor's designee (e.g., a contract research organization). These monitors verify that the rights and well-being of human subjects are protected; that the conduct of the trial is in accordance with the protocol, regulatory requirements, and good clinical practices; and that data reporting (including safety reporting to IRB, FDA, and NIH) is accurate and complete.

In addition, in those instances where the gene therapy trial has an independent data and safety monitoring board (or equivalent) associated with it, the board's findings and recommendations regarding patient safety are shared with the IRB, FDA, and NIH. In some gene therapy trials, one or more of the investigators is also the sponsor or a member or employee of the sponsoring organization. NIH will work to develop procedures to further assure appropriately independent oversight of the conduct of such trials.

"Clinical trial monitoring and responsible reporting must be taken seriously by all parties involved in gene therapy trials," said Commissioner of Food and Drugs Jane E. Henney, M.D. "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 will take place during this week's meeting of the Recombinant DNA Advisory Committee (RAC). Scientists and physicians will discuss the safety and future clinical applications of a new class of adenoviral vectors that have been extensively altered with the aim of improved safety.

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New Initiatives to Protect Participants in Gene Therapy Trials