バイオ研究者・大学院生のための簡単な英語文例集　その２３

その８で紹介したガイドラインの改正案に関連したニュースが雑誌 (FDA to release data on gene therapy trials. in Science 291: 572-573,2001) に載っております（オリジナルのScienceの記事を参照。）ので以下に紹介します。

CLINICAL MEDICINE:
FDA to Release Data on Gene Therapy Trials

Jocelyn Kaiser

Moving to allay public concerns over the risks of gene therapy experiments, the U.S. Food and Drug Administration (FDA) last week proposed publicly releasing much of the safety and protocol data from clinical trials that it now keeps confidential. The agency wants to apply the same policy to animal-to-human transplants, another controversial experimental procedure.

Several gene therapy researchers praised the decision. "We think public fears should be assuaged, and one way to do it is to make the information available," says Inder Verma of the Salk Institute for Biological Studies in La Jolla, California, president of the American Society of Gene Therapy. Phil Noguchi, director of the cellular and gene therapy division at FDA's Center for Biologics Evaluation and Research, agrees that the proposed rules are important symbols: "It's the perception of something being hidden that's the scary part." Biotech industry officials, however, are not pleased; they worry that releasing clinical data could stifle drug development and that the public may misinterpret the safety reports.

The changes come in response to the 1999 death of 18-year-old Jesse Gelsinger in a gene therapy trial at the University of Pennsylvania in Philadelphia. The incident triggered a flurry of reports and congressional hearings on whether safety problems from this and other trials were being fully disclosed by sponsors, whether academic or commercial (Science, 12 May 2000, p. 951). It also revealed the confusion over current government reporting requirements.

Under the proposed rule, FDA would make public much of the information that sponsors now submit in confidence to the agency on their gene therapy clinical trials, including preclinical toxicity data, protocols, informed consent forms, ongoing reports of adverse events, and records of any FDA investigations. Under FDA rules, for example, companies must report within 7 to 15 days serious events that are unexpected and possibly related to the therapy. Companies themselves would remove personal and confidential business information from these documents, which FDA would then post on the Internet.

The Biotechnology Industry Organization (BIO) says the FDA proposal sets a "troubling precedent" by carving out an exception to its confidentiality policy. BIO officials are concerned that confidential patient and business data may inadvertently be released. What's more, says BIO bioethics counsel Michael Werner, releasing data on all adverse events before they can be investigated could "be misleading or misunderstood or taken out of context" by patients and the public, as many of these problems are related to a patient's underlying disease and not the therapy. But Noguchi disagrees, noting that the only events that sponsors have to report immediately are those possibly related to treatment; the rest are summarized in an annual report.

Federal officials say this new body of information will "complement" the work of the Recombinant DNA Advisory Committee (RAC), which advises the director of the National Institutes of Health (NIH) on the ethics and safety of gene therapy trials. The RAC already releases protocols and safety reports on NIH-funded trials (www4.od.nih.gov/oba/rdna.htm). (Those few investigators with no direct or indirect NIH funding can submit information voluntarily to the RAC but would be obliged to follow the FDA rule.) The RAC now wants to analyze those adverse event reports for trends and recently proposed establishing a new working group to do so. Amy Patterson of the NIH Office of Biotechnology Activities, which runs the RAC, explains that the FDA proposal will satisfy the public's desire for access to safety information right away, while the RAC will continue to provide an open forum for analyzing the reports.

The rules also cover the rapidly evolving field of animal organ and tissue transplants. FDA plans to release data from such xenotransplantation trials, while the Department of Health and Human Services (HHS) is finalizing more stringent guidelines for trials as part of a broader effort to reduce the risk of introducing new viruses into the population (Science, 30 January 1998, p. 648). A new HHS xenotransplant advisory committee--similar to the RAC--will hold its first meeting in late February.

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