Changes in Gene Therapy Rules Debated
During an often confusing and emotional debate at a packed National Institutes of Health conference, scientists, health officials, ethicists and biotech representatives differed sharply Friday on how to change existing NIH guidelines for the troubled field of gene therapy research.
Although no clear consensus emerged, several scientists and officials said significant changes are necessary to restore public trust--a trust shaken by the first reported gene therapy-related death and by requests from researchers and sponsors to keep serious problems in gene therapy research confidential. Such a confidential designation would prevent public review of gene therapy techniques, some scientists say.
“We’re in a listening mode right now,” said Lana Skirboll, director of the NIH’s Office of Science Policy and an advisor to NIH director Harold Varmus. But, she told reporters, “there is no doubt that the guidelines will be clarified to reflect public concern.”
The public meeting of the NIH Recombinant DNA Advisory Committee followed two days of hearings aimed at discovering why 18-year-old Jesse Gelsinger died during a University of Pennsylvania gene therapy trial in September. Gelsinger, who had a genetic liver disorder called OTC deficiency, died after receiving a weakened cold virus, called an adenovirus, used as a vehicle to deliver new genes. FDA officials have said the team violated several protocol and reporting guidelines, though no evidence has emerged to suggest that their actions directly caused Gelsinger’s death.
But even before the tragedy, NIH officials had expressed concern about a new trend. Biotech researchers, intent on protecting trade secrets, had begun asking the Recombinant DNA Advisory Committee to keep confidential their reports detailing so-called adverse events or side effects seen during clinical trials. Under federal regulations, an experiment’s sponsors must report such events to the FDA within 15 days of receiving the information and within seven days if the serious adverse event is deemed life-threatening or results in death.
“The NIH does not consider adverse events to be trade secrets, period,” Skirboll said. But she confirmed that under current NIH guidelines “people are allowed to mark things secret--and they have.”
Troubled by the new trend toward secrecy, the NIH urged the advisory committee to study how to effectively remove the loophole. The committee has also proposed a clarification of often-confusing language about what constitutes an adverse event, which ones should be publicly reported and when that reporting should take place. A final proposal would require that patient identification be removed from these reports to ensure privacy.
But many scientists, biotech officials and even some of the advisory committee’s members argued that the additional guidelines would be burdensome. Biotech officials said the FDA already oversees gene therapy trials and maintained that more NIH rules would add another confusing layer of bureaucracy. Robert Spiegel, a senior vice president and medical officer for the Schering-Plough Research Institute--one of the biotech companies that requested confidentiality--complained that the new guidelines would create a reporting burden that “would obscure rather than highlight a meaningful adverse event.”
In emotional testimony, however, former advisory committee member Karen Rosenberg chastised the panel for becoming little more than “a public debating society” and denounced biotech officials. “The future of gene therapy has to be based on trust from the public,” she said.
In 1996 Varmus stripped the advisory committee of its regulatory authority, transferring that power to the FDA. But last week he announced the formation of a committee to review the role of the NIH in overseeing clinical gene therapy research.
