Print“Downing and his co-authors suggest that the FDA could work harder to produce straightforward evaluations of these trials for patients and caregivers.”
Flexible U.S. Food and Drug Administration approval standards intended to bring lifesaving drugs to market faster also necessitate closer monitoring and evaluation of those drugs in the market, suggests a study of agency review data.
Although the majority of FDA-approved indications were supported by at least one pivotal, randomized, double-blinded trial, there was wide variation in trials’ choice of comparators and end points, duration, size, and completion rate, according to the study, published in the Journal of the American Medical Association.
The variation may make it difficult for patients and doctors to assess the benefits, safety, and efficacy of medications for new indications, suggests Nicholas Downing, a medical student at Yale University School of Medicine and the study's lead author. Comparative effectiveness information was available for less than half of new indications approved during the study period, making it even more difficult for clinicians and patients to compare new options to other medication on the market.
“Because comprehensive safety evaluations are difficult to undertake as part of randomized controlled trials, particularly smaller trials,” wrote Downing, it’s important to adopt “a 'life-cycle' approach, both for drug safety and for improved understanding of drug effectiveness.”
The study reviewed FDA approvals of 188 novel therapeutics for 206 indications. The approvals, awarded between 2005 and 2012, were made on the basis of 448 pivotal efficacy trials. Though most indications approved were supported by two pivotal trials, 74 indications were approved solely on the basis of a single pivotal trial. The ability to make approval decisions based on single a pivotal trial and to use surrogate end points in trials when they are reasonably likely to predict clinical benefits are hallmarks of the agency’s effort to speed the process of bringing lifesaving drugs to market. But Downing and his co-authors suggest that the FDA could work harder to produce straightforward evaluations of these trials for patients and caregivers.
The study proposes the creation by FDA of a summary statement updated throughout a drug's commercial lifetime or a letter grade for each approval signaling the quality of the clinical trial evidence used to determine its safety and efficacy.
Just as the FDA has worked to encourage drugmakers to embrace innovative and less burdensome trial designs to make clinical trials more efficient, Downing writes, "it also must ensure that patients and physicians understand how to interpret the results of these trials and the likelihood of experiencing benefit or harm when deciding to use these newly approved agents."
January 23, 2014
http://www.burrillreport.com/article-vigilance_called_for_as_fda_speeds_approvals.html
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