Published On July 23, 2008
ANYONE WITH A LIFE-THREATENING DISEASE HOPING TO KEEP UP with research into her condition is at several disadvantages. To learn the outcome of a clinical trial, she can read about it only in a journal, and because there are too many research papers for too few journals, many studies go unpublished. What’s more, if the study’s results are negative, drugmakers who sponsored the trial might choose not to publish the results or might even suppress them, as was allegedly the case with Vioxx, a pain reliever that was voluntarily withdrawn from the market in 2004.
To remedy this lack of transparency, the United States will become the first nation to launch a database of results of clinical trials that involve drugs, medical devices and biologics (products derived from living sources, such as vaccines). The data will be available online within one year of a study’s completion or within 30 days of Food and Drug Administration approval. When the database debuts on September 27, it will include trials that were under way as of or after Sept. 27, 2007. (Researchers who conducted trials of drugs that have been on the market for years are encouraged, but not required, to post their results.) The law threatens significant penalties—including withholding of federal funding and up to $10,000 per violation—for drugmakers and other researchers who fail to submit the required data.
Trial results, except for those involving Phase I investigations (which determine the basic safety and dosage range of a drug or product, and which were left out because of political compromise and practical considerations, such as protecting researchers’ intellectual property rights), will be posted at ClinicalTrials.gov. The site now records such details as study design and outcome measures of more than 56,000 trials but no results.
The database is good news in at least one respect, says Bob Helms, founder of Guinea Pig Zero, a job zine for trial participants. Under the new law, sponsors will find it harder to conceal unexpected or dangerous reactions to drugs that could put future volunteers at risk. But Helms warns of a possible unintended consequence: more exporting of the clinical trials industry. Trial sponsors “might take research units to places like India and Russia, where the rules are almost mythical, if they exist at all,” he says. Deborah A. Zarin, director of ClinicalTrials.gov, confirms that if a trial is conducted entirely outside the United States and the drug under study is not manufactured here, the FDA would have no regulatory authority.
Critics question how the lay public will be able to interpret results that haven’t been peer reviewed and thoroughly discussed in medical journals. On the upside, Zarin notes, the structure of the results database—with fill-in fields and little room for commentary—will keep spin to a minimum.
Researchers’ responsibilities will expand in September 2009, when they must begin reporting serious and frequent adverse events. And by the following year, the Department of Health and Human Services will have to develop regulations to further expand the database, including full protocols (such as a summary of the study’s purpose) or additional information to better evaluate results.
Meanwhile, the movement toward greater research transparency is gaining converts around the globe, and the World Health Organization recently conducted a survey to help craft a global standard for publicizing trial results. Zarin says, “The rest of the world could benefit by observing the effects of this national experiment.”