GENE THERAPY: FDA and NIH Roll Out New Monitoring Rules
To restore confidence in gene therapy trials, the FDA and NIH announced a new set of initiatives "designed to enhance the safety of patients involved in gene therapy experiments," the Washington Post reports. Although gene therapy researchers are already required to institute safety monitoring plans, they now will have to submit those plans to the FDA for review before experiments take place. Experiment sponsors will have to hire "specially trained individuals whose job will be to ensure that patients' rights are protected, the studies are conducted as approved by the FDA and all safety data such as side effects are reported properly to university and federal officials." The FDA and NIH will hold joint quarterly meetings where gene therapy researchers can exchange information from their studies, including safety reports.
A Necessary Step
FDA Commissioner Jane Henney said, "Clinical trial monitoring and responsible reporting must be taken seriously by all parties involved in gene therapy trials. Our plan will help restore the confidence in the trial's integrity that is essential if gene therapy studies are to be able to fulfill their potential" (Weiss, 3/8). Kathryn Zoon, director of the FDA's Center for Biologic Evaluation and Research, added, "We believe that these are important steps ... to ensure patient safety." Zoon said that if the FDA did not like a particular monitoring plan, the agency could "put them on hold." She said that the FDA would assess whether such standards would work in other fields of medical research (Fox, Reuters/Contra Costa Times, 3/8). FDA officials also revealed that the agency had sent letters to researchers "demanding additional assurances that the viruses and cells used in gene therapy treatments were free of contamination." The agency plans to propose a new rule to allow it to "publicly disclose certain details about the safety of gene therapy experiments" -- information that currently is kept secret by law.
Cheers and Jeers
Michael Werner, bioethics counsel for the Biotechnology Industry Organization, said that the biotech industry supports the initiatives, saying, "It's important the public knows this research will be conducted responsibly. Our goals and the oversight agencies' goals are the same" (Washington Post, 3/8). A spokesperson for Rep. Henry Waxman (D-Calif.), a vocal critic of gene therapy oversight, also commended the new initiatives: "These are precisely the kinds of activities that should have been done earlier." But Alan Milstein, an attorney representing the father of Jesse Gelsinger, who died in a University of Pennsylvania gene therapy experiment, said, "The FDA and NIH have yet to address their mistakes with respect to Jesse's death -- particularly and most glaringly their approval of Penn's study, which had the prospect of little benefit and tremendous risk for patients" (Collins, Knight Ridder/Contra Costa Times, 3/8).