By Kim Coghill
WASHINGTON - The FDA said its proposal to require public disclosure of certain data and information related to human gene therapy and xenotransplantation is a move to better protect and inform the public of unique risks associated with these types of research.
But the Washington-based Biotechnology Industry Organization (BIO) doesn't believe the agency's motives are that cut-and-dried.
"The FDA has been kicking this around for a while, but obviously they felt the time was right now to release it," said Michael Werner, BIO's director of federal government relations/bioethics counsel. "It means a dramatic deviation from about 30 years of FDA regulation and practice to release clinical trial data to the public."
Under the proposed amendment, sponsors of clinical trials on human gene therapy or xenotransplantation would be required to submit to the FDA a list of details related to the investigational new drug application, including product and patient safety data, the protocol for each planned study, and identification of the biological products and a general description of the method of production.
Human gene therapy and xenotransplantation are being targeted because they have the potential of infecting the patient and potentially the public, and also due to modification of the human genome, the FDA said.
Werner said gene therapy has come under increased scrutiny since the September 1999 death of Jesse Gelsinger, 18, a University of Pennsylvania gene therapy patient. Gelsinger was a participant in a Phase I gene therapy study for patients with ornithine transcarbamylase deficiency. He died days after receiving a hepatic infusion of an altered adenoviral vector.
"I think the FDA is trying to respond to some political pressure and we have said the FDA should have the resources to enforce existing rules and that they should do everything they can to protect patient safety," Werner said. "They already have the responsibility and right to put a trial on hold or shut down a trial if they think patients are at risk, but releasing data from clinical trials is just inappropriate."
The FDA said much of the information already is being released on a voluntarily basis through the Internet, and oftentimes company web sites provide this type of information in descriptive summaries of clinical trials, press releases, recruitment for patients, investment opportunities and general awareness material.
Thus, the FDA rationalized, the information already is public and its disclosure has not impeded commercial development.
On the contrary, Werner said, data from the clinical trials could be misleading or subject to misinterpretation, "and potentially, proprietary information would be made public. And we are concerned that the FDA would eventually apply this rule to all clinical trials."
Furthermore, Werner said the proposal requests information that is not currently being disclosed to the Bethesda, Md.-based National Institutes of Health, including the results of product safety tests and the description of the method of production.
The FDA said confidential commercial information (suppliers or trade secret manufacturing information), names of patients and their third-party contacts (physicians or hospitals), will not be disclosed.
Human gene therapy and xenotransplantation are being proposed to treat genetic diseases such as cystic fibrosis, cardiovascular insufficiency, metabolic diseases such as diabetes, neurologic diseases such as Parkinson's and Huntington's disease, cancer, acquired immune deficiency syndrome and organ failure.
Under the rulemaking process, there will be a 90-day public comment period. Written comments may be submitted to: Dockets Management Branch; Food and Drug Administration; 5630 Fishers Lane, Room 10-61; HFA-305; Rockville, Md. 20852.
The full report can be viewed on the FDA's web site at www.fda.gov.