Soon after the Food and Drug Administration Amendments Act of 2007 (FDAAA) took effect, tightening new-drug reviews with the goal of preventing approval of blockbuster drugs with deadly side-effects like Vioxx and Celebrex, the biopharma industry began years of complaining about FDA red tape slowing down the pace of new-drug decisions—to  the detriment, they said, of patients.

But a study published today by JAMA Internal Medicine questions that widely cited premise, raising the issue of whether safety has been sacrificed by a provision of the FDAAA allowing expedited approvals of drugs deemed to offer significant therapeutic advancement for “serious or life-threatening conditions that would address an unmet medical need.”

Thomas J. Moore, A.B., of the Institute for Safe Medication Practices—a nonprofit dedicated to medication error prevention and safe medication use—and Curt D. Furberg, M.D., Ph.D., of the Wake Forest University School of Medicine, studied development times, clinical testing, post-market follow-up, and safety risks for the 20 drugs approved by the FDA in 2008.

Eight of the 20 drugs approved in 2008 were approved via expedited review; the rest, under standard review. Expedited drugs took a median of 5.1 years of clinical development to win FDA marketing approval, compared with 7.5 years for drugs approved through standard review, according to the study results.

Dr. Furberg and Moore found that far fewer patients were tested for drug efficacy during expedited approvals—a median of 104 patients, compared with a median 580 for standard-review drugs. More sobering: As late as this year, many postmarketing studies requested by the FDA to gather additional evidence on the safety of expedited drugs had not been completed, according to the researchers.

The safety concern, according to Dr. Furberg and Moore, reflects a consequence of the expedited approval review process: Most testing of such drugs occurs after small trials in more narrowly defined patient groups, followed by extensive additional testing once those treatments win FDA approval.

“Our findings suggest that the shift has made it more difficult to balance the benefits and risks of new drugs. Further systematic assessment of the standards and procedures for testing new drugs is needed,” the authors concluded.

In a related commentary published in the same issue of JAMA Internal Medicine, Daniel Carpenter, Ph.D., of Harvard University, observed that the findings of Dr. Furberg and Moore “underscore the continuing importance of rigorous premarket studies of ample size.”

“If the critical phrase ‘serious or life-threatening conditions that would address an unmet medical need’ is defined broadly enough (and there are lobbying efforts to define it as broadly as possible), the future of evidence for pharmaceuticals in the United States will look more like 100 patients for efficacy trials instead of 500 patients,” wrote Dr. Carpenter, who in 2011 called for the FDA to be an independent agency insulated from political pressures by removing it from the Department of Health and Human Services.

Dr. Carpenter concluded: “If the FDA’s requirements for new drugs, both premarket and postmarket, are weakened, trust in both the efficacy and safety of prescription drugs is likely to be weakened. The stakes of the current policy debates could not be higher. There is scarcely a feature of the American healthcare system that does not depend on evidence-based trust in prescription drugs, ratified and enforced by the FDA.”

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