Panel proposes framework for FDA to evaluate drug risks

The withdrawal of the painkiller Vioxx and other drugs from the market highlights a central challenge faced by regulatory authorities: how to ensure that life-saving therapeutics are available in a timely fashion while still guaranteeing the medicines are safe. It's a difficult task, but one that might be made easier with a more centralized and comprehensive system for drug safety monitoring, according to the US Institute of Medicine (IOM), an independent, nongovernmental organization in Washington, DC.

Under a proposal outlined by the IOM last month, the US Food and Drug Administration (FDA) would compile risk assessments, evidence about adverse side effects, relevant studies and regulatory actions related to each drug on the market in a single, publicly accessible document known as a benefit-risk assessment management plan (BRAMP). (At present, such information is scattered throughout the agency, making it difficult for consumers and health providers to readily access it.) The IOM report also calls for therapies approved on the basis of trials that used surrogate endpoints such as blood pressure, rather than clinical endpoints such as heart attacks, to receive extra scrutiny after they reach pharmacy shelves.

“We should be looking at drugs during their entire market lifetime, with the intensity of scrutiny based on the potential for patient or population harm, periodically revisiting them to make sure there haven't been changes in the risk profiles,” says Steven Goodman, co-chair of the IOM committee and a biostatistician at the Stanford University School of Medicine in California.

Current FDA pharmacovigilance efforts don't do a good enough job of anticipating risk, leaving regulators scrambling for information when concerns arise, says Goodman, who notes, “We can't wait for the fires to put them out because there are few good—or fast—options in a moment of crisis.”

In a statement, the FDA said that the IOM report mirrored the agency's current programs but that implementing a public repository would pose a financial challenge that might compromise other “critical regulatory activities.” FDA officials would not elaborate on what recommendations from the report it might implement or how. “We are now in the process of fully analyzing the report to determine how to implement changes that will have the largest positive impact on public health,” Sandy Walsh, FDA spokeswoman, wrote in an email to Nature Medicine.

But even if the agency does adopt the IOM plan, that won't completely eliminate problems, says Michael Carome, deputy director for health research at Public Citizen, a research-based advocacy group in Washington, DC. “These recommendations won't [protect against] drugs that end up having serious safety problems from reaching the market because [their] premarket studies were small and not sufficiently powered to detect uncommon or rare safety problems,” he says.


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Stokes, T. Panel proposes framework for FDA to evaluate drug risks. Nat Med 18, 839 (2012).

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