By Kim Coghill
BETHESDA, Md. - Members of an FDA advisory committee spent Friday afternoon trying to balance the right of trial sponsors to keep certain data confidential and the public's right or need to have access to that information.
In the final hours of a two-day Biological Response Modifiers Advisory Committee (BRMAC) meeting, members discussed the FDA's proposed rule for public disclosure of data and information related to human gene therapy or xenotransplantation. The FDA released the proposal in mid-January and will accept comments through April 18. (See BioWorld Today, Jan. 19, 2001.)
The regulation is specifically for human gene therapy and xenotransplantation because these are the areas of clinical research that have the potential for public health risks and modification of the human genome.
"Part of the need for a rule is that these are products of nature and in nature there are no rewards or punishments, only consequences," Philip Noguchi, the FDA's director of the Division of Cellular and Gene Therapies, told the committee.
And although the 1999 death of Jesse Gelsinger, a gene therapy patient in a University of Pennsylvania study, didn't prompt the agency to consider the rule, the incident illustrates the consequences and potential threat of testing drugs and therapies, Noguchi said.
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.
The proposed regulation requires disclosure of information from the investigational new drug application (IND) including: product and patient safety data and related information including results from preclinical and clinical studies and tests that demonstrate the safety and/or feasibility of proposed procedures; the name and address of sponsor; the clinical indications to be studied; the protocol for each planned study; written consent forms; identification of the biological product(s) and a general description of the method of production, including features that may affect patient safety; IND safety reports; information submitted to FDA in the annual report; and regulatory status of the investigation.
The regulation does not require disclosure of patient information, trade secrets and confidential commercial information.
Noguchi said the proposal is a "radical change in regulation," and that public disclosure of information such as adverse events is not necessarily a bad thing. "The public can benefit when we report adverse events because we can learn from them," he said.
Abbey Meyers, a consultant to the board and president of the National Organization for Rare Disorders in New Fairfield, Conn., and consultant to BRMAC, said, "From the patient standpoint, this regulation is one of the most important things the FDA has done. There seems to be a lot of secrecy in trials. The only time something is reported is when Wall Street gets it and the media picks it up and writes about it."
She said information about adverse events should be available on the Internet for families to review before they determine whether they want to participate in a trial. "The Gelsinger family didn't have a fighting chance because they had no idea about the animals who had died and if we don't put this information out there - it ends up on '60 Minutes.'"
But other board members argued that data from clinical trials could be misleading or misinterpreted.
Alison Lawton, senior vice president of regulatory affairs at Cambridge, Mass.-based Genzyme Corp. and a nonvoting member of BRMAC, said, "We recognize the need for public debate, but is this rule the right way to do it? My perception is no."
She also said the committee should consider other ways of disseminating information, and not necessarily through the Internet.
Michael Werner, director of federal government relations/bioethics counsel for the Washington-based Biotechnology Industry Organization, said there is a misperception that the information the FDA wants disclosed is voluntarily being released. He advises against releasing the information from an IND, saying it could end up causing serious problems for companies trying to develop products.
"Simply put, the premature release of information makes it more difficult to develop products and ultimately the patient suffers," he said. "This proposal represents a dramatic and troubling change in FDA policy."
Human gene therapy is defined as the administration of genetic material to modify or manipulate the expression of a gene product or to alter the biological properties of living cells for therapeutic use.
Xenotransplantation refers to any procedure that involves the transplantation, implantation or infusion into a human recipient of either live cells, tissues or organs from a nonhuman animal source; or human body fluids, cells, tissues or organs that have had ex vivo contact with nonhuman animal cells, tissues or organs.
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, AIDS and organ failure. n