Sept. 13, 2000 (Washington) -- The chief advocate of this nation's state of health, Health and Human Services Secretary Donna Shalala, is telling American researchers in no uncertain terms that they must go the extra mile to protect patients in research experiments -- or risk losing public support for these critically important clinical trials.
"To put it simply, if we cannot guarantee sound research in general -- and patient safety in particular -- public support for gene therapy and other potentially lifesaving treatments will evaporate," writes Shalala. The National Institutes of Health budget alone contributes some $15 billion annually in research support.
Shalala's strongly worded comments, which come in a Sounding Board commentary in the Sept. 14 issue of TheNew England Journal of Medicine, reflect a growing concern following the death of a young patient in a gene therapy trial last year and the subsequent closing of several clinical trials since.
Shalala singled out the fate of 18-year-old Jesse Gelsinger, who became the first person to die because of a gene therapy treatment, in this case for a rare liver disorder. According to Shalala, Gelsinger and others who received the replacement gene in a clinical experiment at the University of Pennsylvania were not given appropriate protections, nor were they apprised about "the appearance of a substantial [financial] conflict of interest" involving the researchers and trial sponsors. Also of concern, writes Shalala, are hundreds of unreported "adverse events" -- some life threatening -- experienced by gene therapy study volunteers.
In light of these recent happenings, research institutions must take the lead in responding to experiments that subject people to possible harm, Shalala contends. That might even include "remedial action" on the grounds that the highest research priority is not advancing knowledge, but protecting those patients who take part.
Meanwhile, the National Bioethics Advisory Commission, created in 1995 to protect those who participate in human trials, is meeting here this week to finalize a draft proposal for improved ethical standards underpinning clinical trials. Commission chair and Princeton University president Harold Shapiro, PhD, praised Shalala's blunt comments.
Shalala's comments, says Shapiro, could help enhance the significance of patients in clinical trials. "Yes, it's a little more [time-consuming] to treat people as if they're really, really important, ... but the benefits are so great for everybody, including investigators, that prioritizing a patient's safety ought to be a very simple decision," Shapiro tells WebMD. Shapiro also agrees that it is possible to advance biomedical research and ethics simultaneously.
Shalala goes on in her piece to lambaste dubious or misleading efforts to recruit patients into "risky" experiments. Suggesting that people will benefit without giving them the whole picture is "unconscionable," she writes. Other worries include allowing people into experiments when they don't meet the proper criteria and putting too much strain on institutional review boards. These oversight committees, which exist at each research institution, often lack sufficient resources to handle their substantial responsibilities, says Shalala.
"Given this state of affairs, the federal government, researchers, and research institutions must come together with a sense of urgency to reform the current system," writes Shalala. That process, she says, is already under way with new training and enforcement programs. On-site monitoring of experiments is also being enhanced, and data and safety monitoring boards, independent panels of experts who help monitor the progress and safety of clinical trials, provide another layer of supervision.
If Congress passes enabling legislation, the FDA could be given the authority to fine institutions that violate research ethics up to $1 million. In addition, an upgraded Office for Human Research Protections will set a tough enforcement standard. This office, which was established in June, counsels the Department of Health and Human Services on how to best protect the interests of patients.
A spokesperson for Shalala said the secretary -- in an effort to reach the medical community -- had planned to write a journal article in May when the Department of Health and Human Services announced these new federal ethics guidelines. However, it's taken several months for final publication.