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Companies question FDA disclosure rules By Ronald Rosenberg, Globe Staff, 1/31/2001 iotechnology companies planning to conduct gene therapy and animal- to-human transplants are questioning new regulations that require them to provide more details about their tests and immediate public notification of side effects. The proposed regulations from the US Food and Drug Administration are designed to balance patient advocates' call for more public disclosure of controversial gene therapy trials with biotech companies' desire to keeping data confidential for competitive reasons. The FDA said the new rules will provide public access, through the Internet, to a gene therapy clinical trial's design and safety parameters but without confidential personal information about the participants. In a gene therapy trial, where copies of normal genes are used to replace or offset malfunctioning genes in a patient, researchers would have to publish the criteria for choosing participants, precisely what is being tested, and any serious side effects. The same rules apply to xenotransplantation, the use of live cells, tissues, or organs from animals that are transplanted into humans. The public has 90 days to submit comments on the proposed new rules. After that, the FDA will issue the final regulations. But companies have already begun expressing their opposition. ''There seems to be a lot more politics than science in these rules,'' said Michael Astrue, chairman of the Massachusetts Biotechnology Council and general counsel at Transkaryotic Therapies Inc., both in Cambridge. Astru e called the rules ''horribly unfair.'' But consumer advocates praise the FDA change as a necessary and long-overdue way to help ailing Amercians make better decisions before participating in medical experiments like controversial gene therapy clinical trials. ''The real fact about gene therapy is that scientists and the companies sponsoring these studies know no one is being cured, yet the public is clamoring to become involved,'' said Abby Myers, executive director of the Nat ional Organization for Rare Diseases in New Fairfield, Conn. ''But new technologies are evolving, so to earn the public's trust will require more reassurance and disclosure.'' Released earlier this month, the new FDA rules were sparked by the 1999 death of Jesse Gelsinger, an 18-year old from Tucson. His death from a liver disorder stemming from the failed gene therapy treatment led to the discovery that many researchers were not following guidelines by the National Institutes of Health. The government funding agency requires scientists to immediately report so-called adverse events, such as deaths, life-threatening episodes by participants, and significant changes in patients' health. Last February, gene therapy programs at St. Elizabeth's Medical Center in Brighton were abruptly halted after federal regulators charged Dr. Jeffrey M. Isner with lapses in reporting two patient deaths and using medically ineligible patients. These incidents prompted an outcry for more public disclosure to the National Institutes of Health. The agency oversees gene therapy and xenotransplantation studies conducted with federal funds. And while the FDA decides whether scientists can try experimental therapies, participants are supposed to receive consent forms detailing all the potential risks. However, until now, the FDA rules permit companies to with hold details of gene therapy trials because they are considered commercially important trade secrets. The new FDA rules will require more public access to ongoing gene therapy and xenotransplantation clinical results. ''Both of the technologies hold great promise, but they may also pose a remote but unique risk to the individuals who have volunteered to participate in these types of studies,'' FDA commissioner Jane E. Henney said in a statement. ''Our proposal will ensure that the public is fully informed as we investigate these new public health opportunities and challenges.'' Not everyone agrees. Some biotech companies maintain the new rules will lead to confusion over whether a study is truly unsafe and potential misunderstanding of whether a participant's side effects were due to the underly ing diseases or the gene therapy. And some companies maintain the rules will impact their businesses unfairly. ''If our entire clinical design is out in the public domain, along with individual results, it means that competitors can watch what we are doing, design their own systems, and get around what we have worked so hard for,' ' said Julia Greenstein, president of Imerge BioTherapeutics Inc. of Charlestown, a recently formed joint venture between her former employer, BioTransplant Inc., and Novartis Pharma AG. Imerge is focused on providing swine-based cell tissues and organs that can be transplanted into humans and provide a lifeline for people waiting for organ transplants. And while initial clinical trials are still several years away, Greenstein said she understands the need for public debate as well as business confidentiality. ''Frankly, the new rules are not very clear,'' she added. Ronald Rosenberg can be reached by e-mail at rosenberg@globe .com. This story ran on page C4 of the Boston Globe on 1/31/2001. © Copyright 2001 Globe Newspaper Company. http://www.boston.com/dailyglobe2/031/business/Companies_question _FDA_disclosure_rulesP.shtml [log in to unmask]