FDA Proposes New Rules for Gene Therapy Trials
In a "major" policy shift, the FDA yesterday issued a proposed new rule that would lift the "cloak of secrecy" from gene therapy and xenotransplantation -- transplanting animal cells into humans -- experiments, the Philadelphia Inquirer reports. Under the new regulations, the FDA would provide the public with information about the safety and progress of the "highly experimental" trials on the agency's Web site (Collins, Philadelphia Inquirer, 1/18). The proposed rule calls for the FDA to release "full descriptions" of all gene therapy and xenotransplantation clinical studies, the results of safety tests leading to the studies, copies of participants' consent forms, descriptions of monitoring procedures, a "constantly updated" record of safety problems and a record of FDA disciplinary action. "Today's action is an important step in ensuring greater public confidence in these revolutionary therapeutic technologies," FDA Commissioner Jane Henney said (Weiss, Washington Post, 1/18). "Both of these technologies hold great promise, but they may also pose a remote but unique risk" to study participants she added (Adams, Wall Street Journal, 1/17). Dr. Kathryn Zoon, the FDA's biotechnology chief, said, "The more transparent the process is ... the more public acceptance and public confidence there will be" (Associated Press, 1/18). According to the FDA, the new rule would have "little practical impact" on the biotechnology industry because companies already report most of the information on such trials to the NIH. In addition, the regulations would still allow firms to "withhold" confidential or "commercially sensitive" information and patients' medical records. The FDA will accept public comment on the proposal for 90 days (Washington Post, 1/18).
The proposal comes in the wake of "widespread concern" about the "adequacy" of FDA oversight of gene trials after the 1999 death of 18-year-old Jesse Gelsinger, a participant of an experiment at the University of Pennsylvania (Philadelphia Inquirer, 1/18). "I think the FDA has seen the light here. I hope the politics and the money pressure doesn't undo it," Paul Gelsinger, Gelsinger's father, said. "They don't have to tell their secrets, but they ought to tell about their side effects," Alan Milstein, Gelsinger's attorney, added. Patient advocacy groups and gene therapy researchers have also praised the proposed FDA rules. "I think it's appropriate, and I think it will be positive," Ronald Crystal, director of the Institute of Genetic Medicine at Weill Cornell Medical Center in New York, said (Washington Post, 1/18). He added, "The fact is that gene therapy is remarkably safe, but the public is concerned because they don't know that. The only way we can convince them is through public disclosure" (Wall Street Journal, 1/18). Dr. Sidney Wolfe of Public Citizen called the FDA proposal "terrific" and a "major step forward," but added that the agency should expand the rules to cover all experiments (Associated Press, 1/17). Abbey Meyers, head of the National Organization for Rare Disorders, said, "We've been fighting for this for 10 years. The FDA's biggest problem has always been secrecy" (Philadelphia Inquirer, 1/18).
However, biotechnology firms, hoping to "study and commercialize" the two "cutting-edge" medical technologies, have voiced concerns about the new FDA rules. Michael Werner, bioethics counsel for the Biotechnology Industry Organization, called the regulations a "major deviation" from years of "regulation and practice that said commercially confidential information will remain confidential" (Wall Street Journal, 1/18). In addition, Werner "attacked" the proposal as "unnecessary for patients, harmful to the fledgling field of genetic medicine and perhaps illegal." He said, "We want there to be an easing of public concern. But we'd distinguish that from releasing, in real time, data from clinical trials that could be misinterpreted and misused. We don't think the public is served by that" (Washington Post, 1/18). BIO President Carl Feldbaum added that disclosing the information would "unnecessarily frighten" patients and the public because patients' underlying diseases, not the therapy, trigger a "vast majority" of side effects in clinical trials. "Requiring disclosure of information that has no bearing on patients or the public could hinder the progress of innovative drug development," he said (Cimons, Los Angeles Times, 1/18).
In addition to the new gene therapy trial regulations, the FDA also proposed rules yesterday that would "strengthen scrutiny" of genetically modified foods, the New York Times reports. The new rules would require developers of genetically modified food to subject such food to a safety review before bringing it to market. The FDA also plans to offer the information provided by the companies to the public. In addition, the FDA issued guidelines on how food companies could voluntarily label products as "made without genetic engineering," but the FDA declined requests from numerous consumer and environmental groups to force companies to label all genetically engineered foods. "What any product doesn't need is for there to be suspicion on the behalf of consumers that something is being slipped by them," Henney said. However, many consumer and environmental groups argued that the new rules "do not go far enough." Rebecca Goldburg, senior scientist at Environmental Defense, said, "It's a baby step in what needs to be a leap toward real regulation." The food and biotechnology industries generally "applauded" the rules, hoping that mandatory FDA review will attract consumers that fear genetically modified foods. According to food companies, consumers would "interpret" mandatory labels on foods with genetically modified ingredients as a "skull and crossbones," while a label that a product does not contain genetically modified ingredients could "attract customers" concerned about genetic engineering. "I think you're going to see a number of companies putting on their labels 'nonbiotech,'" Gene Grabowski, a spokesperson for the Grocery Manufacturers of America, said. However, The new guidelines would "impose restrictions" on such labels. The agency said that it would "frown on" the use of labels that call a product "free" of bioengineered ingredients, a claim "virtually impossible to guarantee," and calls any label implying that nonbioengineered foods are "somehow superior" to their bioengineered equivalents "misleading" (Pollack, New York Times, 1/18).