New plans unveiled to protect gene therapy patients

March 7, 2000
Web posted at: 5:31 p.m. EST (2231 GMT)

WASHINGTON (CNN) -- The Food and Drug Administration and the National Institutes of Health Tuesday announced two initiatives designed to better ensure the safety of patients who take part in gene therapy trials.

The FDA is implementing a Gene Therapy Clinical Trial Monitoring Plan and the NIH is sponsoring a series of Gene Transfer Symposia. Both initiatives are designed to enhance current safety standards for patients enrolled in gene therapy trials.

The safety of patients in clinical trials involving gene therapy has been under scrutiny after one patient died during a gene therapy trial at the University of Pennsylvania last year.

The FDA's plan will require that sponsors of gene therapy trials routinely submit their monitoring plans to the agency.

The FDA also will provide surveillance and inspections to trial sites when deemed necessary, to assess whether monitoring plans are being followed.

Agency spokeswoman Monica Ravelle said the FDA can "show up anytime" to make sure that appropriate guidelines are being followed. The goal is "making sure the patients are protected," she said.

The symposia sponsored by the NIH are meant to "provide critical forums" for the sharing and analysis of medical and scientific data from gene therapy research.

The forums will take place quarterly and are expected to bring together leading experts in gene therapy to publicly discuss medical and scientific data involved in trials.

"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 Ruth Kirschstein.

Critics have said that there is not enough oversight in gene therapy trials and are skeptical that there are many more than the one known death which has been reported as a direct result of gene therapy.

"Clinical trial monitoring and responsible reporting must be taken seriously by all parties involved in gene therapy trials," said FDA Commissioner Dr. Jane Henney.

"Our plan will help restore the confidence in trials' integrity that is essential if gene therapy trials are to be able to fulfill their potential."

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