A quote from the Biotechnology Industry Organization that ran in large type in an article Saturday about the release of information about gene therapy experiments incorrectly combined words from two sentences in the organization's statement. It should have said that the organization stated that data about "adverse events . . . are, by definition, trade secrets and confidential commercial information." (Published 12/14/99)
Federal officials overseeing the field of gene therapy searched in vain yesterday for common ground between drug companies that want to keep details of their experiments secret and advocates who favor a more open airing of the field's recently revealed problems.
On the final day of an emotionally exhausting gene therapy conference at the National Institutes of Health in Bethesda, federal officials wrangled over the difference between "serious" and "severe" side effects, biotechnology company officials pushed for less burdensome regulations, and parents of sick children pleaded for more help from both the regulators and those who hope to profit from gene therapy.
The three-day meeting was prompted by the awkward confluence of two events: the September death of a teenager in a University of Pennsylvania gene therapy experiment, and recent efforts by some gene therapy companies to scale back the amount of information about side-effects they must submit to the NIH.
Researchers and companies testing genetic therapies on people are required by the NIH to release to the public more details of their work than are researchers who test conventional drugs. Those rules were devised to ensure that subtle side-effect trends are noticed more quickly, and to foster public confidence in the novel field that seeks to cure diseases by giving people new genes.
Gene therapy has yet to cure anyone. But as the field has grown from one dominated by academic researchers to one driven by companies with millions of dollars at stake, pressure has built to trim the NIH public disclosure rules and have research results reviewed mostly by the Food and Drug Administration, which keeps such information confidential.
"Virtually every detail about the design, size or status of a clinical trial is of potential competitive value," according to a statement by the Biotechnology Industry Organization (BIO) for the NIH Recombinant DNA Advisory Committee (RAC) yesterday. That includes details of "adverse events," the industry group said, which "are, by definition, trade secrets and confidential commercial information."
BIO board member H. Stewart Parker told officials that biotechnology companies were willing to submit to the NIH some details of the most serious and unexpected adverse events as they occurred but only if the companies and the agency could first agree on how much of that information would be made public. NIH now makes all such information available to the public.
Less serious adverse events should not have to be reported to the NIH at all except in annual summary reports, Parker and other industry representatives said, because those details are too easily misconstrued negatively by the public.
"Immediate disclosure of adverse events is bad science," said an official from Schering-Plough Corp. of Kenilworth, N.J., a company whose recent effort to declare its NIH adverse-events reports "confidential" helped prompt yesterday's reassessment of reporting rules.
Others at the meeting objected to that view, saying so much is still unknown about gene therapy that open sharing of results may help prevent tragedies like the one that befell Jesse Gelsinger, the Arizona teenager who died in September. Even if gene therapy is not more dangerous than other experimental approaches, some said, it is scary enough to the public to warrant extra openness.
"My feeling is it's still a unique field," said W. French Anderson, the University of Southern California researcher who conducted the nation's first gene therapy test in 1990. "Standard medical research doesn't strike the same nerve as changing genes and changing the core of what we are as human beings. It's not a safety issue. It's a public confidence issue. And that will remain until people are really starting to see some cures," perhaps in three to five years, Anderson predicted.
Some RAC members questioned whether the NIH had the right to demand so much data if it didn't have the resources to make sense of it. The agency is years behind in its effort to create an online database of gene therapy trials and adverse events.
Others expressed concern that public reporting could run afoul of new patient privacy regulations under construction. Even a few details in an adverse-event report could inadvertently and illegally give away the identity of a clinical test volunteer with a rare disease. If that's the case, some said, it may be that gene therapy volunteers should be asked to sign a special consent form, which states that they accept the possibility that their identities may become public.
Asked if that extra level of public exposure was appropriate to ask of volunteers, Paul Gelsinger, Jesse's father, acknowledged that some might not be as willing as he and his son were to step into the limelight. "That's a tough one. It's a personal choice," he said. "But I'd advise people to be willing to be public, because it's so important that all the information get out."
RAC officials said they would continue the discussion at future meetings, and would welcome public comments on the issue.