Feb. 2, 2000 (Washington) -- The ongoing saga surrounding the FDA's and NIH's failure to protect the safety of patients in gene therapy trials entered a new chapter Wednesday, when Congress introduced a bipartisan bill that would consolidate the federal oversight of all human research experiments under an independent agency. The announcement was made during a Senate hearing, which examined the issue of whether further federal oversight is needed.
The bill, introduced by Rep. Dennis Kucinich, D-Ohio, would transfer the regulatory authority from NIH's Office for the Protection From Research Risk (OPPR), now known as the Office for Human Research Protection (OHRP), to a yet-to-be created federal agency, whose director would be appointed by the president. OHRP is the agency responsible for protecting the safety of patients in clinical trials. "Congress and the public need clearer lines of authority when it comes to the oversight of human medical research experiments," Kucinich said in a prepared statement.
Industry representatives, while agreeing that the FDA and NIH may have dropped the ball with regards to gene therapy, oppose any such move. Stewart Parker, president and CEO of Targeted Genetics, asks, "Why would you need additional regulations [unless you think the current ones aren't working]?" However, the impetus for further regulation clearly exists, with even the president reportedly expressing his concern through an aide to the NIH's Recombinant DNA Advisory Committee (RAC).
Still, the industry might still find some unconventional -- although somewhat reserved -- allies. There is no need for further regulation, says LeRoy Walters, PhD, director of the Kennedy Institutes of Ethics at Georgetown University. It would not take legislation, he tells WebMD, to simply move OHRP and/or RAC to the office of the Secretary of Health and Human Services (HHS), where they could gather reports of treatment-related adverse events and disseminate the appropriate information directly to the public. "NIH took it away, it could give it back," he says, while pointing out that the same process presently is applied to HIV trials thanks to the lobbying efforts of AIDS patients, who wanted to be kept informed about possible new treatment options.
The problem with the current system is not a lack of regulation, Walters adds, but rather the lack of accountability. The FDA has industry-based reasons to keep this information confidential, and the NIH, as the agency that funds many of these projects, is far too biased, he says. Evidence also exists, he tells WebMD, that neither the FDA or NIH ever wanted to be in charge of OHRP. In a letter to past NIH Director Harold Varmus, MD, Walters says, HHS Secretary Donna Shalala in fact asked Varmus to keep OHRP under the auspices of the NIH after he suggested moving it to the FDA. Her reasoning, Walters says, was that the NIH knew more about gene therapy than the FDA, although it was also clear that under the NIH, OHRP would lack the teeth to take any real sanctions against unscrupulous researchers.
The teeth were also removed from RAC, Walters says, which initially was created to review the protocols of these clinical trials, but has yet to review any protocols since 1997. This has led a number of researchers to largely ignore RAC even though they have been told that any adverse events must be reported to RAC, as well as the FDA. In a private conversation with a member of RAC, he adds, industry representatives indicated that they preferred working with the FDA because its regulations are designed to protect their commercial interests. If adverse events became public knowledge, they allegedly said, their stock price would drop, it would help their competitors avoid making similar mistakes, and it would unmask the secret contents of their potential products.
But this unmasking might now be inevitable at the very least for the industry's credibility. The unreported deaths and adverse events "pose serious ethical questions regarding the risks to patients who participate in these trials and the high financial interests at stake," cautioned Sen. Bill Frist, R-Tenn., during the Senate hearing. And when asked if he still supported genetic research, Paul Gelsinger -- father of Jesse Gelsinger, whose death first exposed the lack of regulatory oversight -- responded, "I've lost all confidence in these men."
But while the oversight system is failing, added Eric Kast, a patient currently participating in a cystic fibrosis gene therapy trial. "Gene therapy must continue to move forward as quickly as possible, as it offers me and the 30,000 others with CF a realistic hope for the ultimate cure. ... Don't let me lose that race, when the finish line might be just around the corner," he pled on behalf of patients with still high hopes for the fledgling industry.
- Congress is trying to change the way the federal government monitors scientific experiments done on people by creating a new independent agency that will take on the responsibility.
- Observers in both industry and research acknowledge there have been recent problems with adverse reactions in genetic studies.
- It is still not certain how the FDA -- or any supervising body -- would be able to enforce the standards that already exist on human experimentation.