New Gene Therapy Reporting Rules Proposed

The Clinton administration yesterday proposed new rules for reporting complications that arise in gene therapy studies, the Washington Post reports. The new system, published in yesterday's Federal Register, seeks a "middle ground" between patient advocates who want public disclosure of all side effects in gene therapy studies; drug companies that are "accustomed to keeping the details of their studies confidential"; and researchers who say they are "overburdened with regulations," the Post reports. While the new rules reaffirm that all adverse events be made public, they appease researchers by "loosening" old NIH requirements that demanded all serious complications, whether the direct result of gene therapy or not, be immediately reported. Instead, the new regulations require researchers to "immediately report to NIH only those serious adverse events believed to have been possibly caused by the treatment." Other problems that researchers deem unrelated to the experimental treatment must be submitted in annual summary reports, which would be analyzed by a new NIH subcommittee "to watch for worrisome trends." The Post reports a rehaul of the NIH rules were "triggered" by three events: Several gene therapy companies demanded that adverse event reports be kept confidential; a nationwide review found that researchers were not reporting adverse events to the NIH; and the death of teenager Jesse Gelsinger, who became the first fatality as a "direct" result of gene therapy.

Groups that would be affected by the changes "quickly found fault with various aspects of the proposed rule," the Post reports. Carl Feldbaum, president of the Biotechnology Industry Organization, "criticized" the public disclosure of reports, saying it would provide "too much public access to commercial data." Meanwhile, patient advocates expressed concern that the new rules give researchers too much liberty in determining how adverse events are reported.

Abbey Meyers, president of the National Organization for Rare Disorders, said, "It's very disturbing that they are going to do something like this, where everyone can decide for themselves." But Paul Gelsinger, Jesse's father, approved of "the extra oversight the new committee would bring." He said that he "didn't mind" that some adverse events would not be reported immediately as long as the system encouraged "better reporting overall." Gelsinger said, "Remember, some of these guys weren't reporting adverse events at all before." The NIH is accepting comments on the proposed regulations through Feb. 10 (Weiss, Washington Post, 12/13).