Openness Urged for Some Clinical Trials

The Food and Drug Administration on Wednesday proposed making information available on all clinical trials involving gene therapy and animal-to-human transplants, including data about deaths or serious side effects.

The proposal stems from the controversy that arose last year over the death of an Arizona teenager during a gene therapy study and subsequent revelations that researchers in that trial and elsewhere had violated federal rules, including notifying federal health officials of safety problems.

The FDA, which shares oversight of such trials with the National Institutes of Health, has never released information about them to other researchers or the public. And although the NIH has released some information, it was under no obligation to do so.

FDA Commissioner Jane E. Henney called the action “an important step in ensuring greater public confidence in these revolutionary therapeutic technologies.”

In gene therapy, genetic material is administered to humans to modify or manipulate genes or to alter the biology of living cells for therapeutic purposes.

Xenotransplantation is the term generally used to refer to any procedure that involves the transplantation, implantation or infusion of animal cells, tissues or organs into humans.

Both approaches “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,” she added. The proposal “will ensure that the public is fully informed” as the work continues.

But Carl B. Feldbaum, president of the Biotechnology Industry Organization, which represents more than 950 biotech companies, academic institutions and biotechnology centers, said that disclosing safety problems “without first investigating the causes could unnecessarily frighten patients and the public.”

The vast majority of side effects in clinical trials are related to the patients’ underlying diseases, not the experimental medicines, he said.

Further, under current rules, “when the cause of an adverse event is linked to the drug being tested, the FDA immediately shuts down the clinical trial,” he said.

“Requiring disclosure of information that has no bearing on patients or the public could hinder the progress of innovative drug development,” he added.

Also, public disclosure could compromise patient confidentiality and raise serious proprietary issues, said Michael Werner, bioethics counsel for the trade group.

“It is a dramatic deviation from decades of FDA regulation and practice,” he said. “There was a reason the rules were the way they were. Changing them would be inappropriate.”

The FDA said its proposal would ensure that the agency’s policies will be consistent with other government agencies, including the NIH. Eventually, much of the information will be posted on the FDA’s Web site.

Gene therapy information is already discussed to some extent at open meetings of NIH’s Recombinant DNA Advisory Committee.

Werner acknowledged that “the NIH makes some information public” but that the FDA proposal will go beyond what the NIH allows. He cited, for example, “descriptions of how [gene therapy] products are produced, toxicology information--all kinds of extra information not routinely submitted by companies to [NIH].”

The proposal will be open to public comment for 90 days before the agency issues a final ruling.