Ellen V. Sigal, PhD

As Capitol Hill prepared for a leadership change following the 2006 elections,  health policy advisers were keenly focused on how the elections would affect the  anticipated debate surrounding drug safety. Within the Washington Beltway, the  Prescription Drug User Fee Act (PDUFA) reauthorization was widely presumed one  of the most important pieces of legislation to receive consideration by the  110th United States Congress.

PDUFA was created in 1992 amid pressure from the HIV/AIDS community about the  Food and Drug Administration’s prolonged drug review time. While PDUFA should  not replace congressionally appropriated dollars, the added resources have  greatly improved overall efficiency of product review. But the recent  identification of potential risks with products like Vioxx (rofecoxib) and  Avandia (rosiglitazone) prompted widespread media scrutiny, reduced public  confidence, and the expectation that sweeping drug safety provisions would be  included in the reauthorization of PDUFA.

Earlier this year, I was among a group of expert scientists, clinicians, and  advocates that issued a report, “Drug Safety & Drug Efficacy: Two Sides of  the Same Coin,” on how to best achieve a responsible balance between safety and  access. In a safety-focused environment, it’s important to understand what’s at  stake.

Scientists and medical professionals acknowledge and accept that drugs are  never 100 percent safe and it’s impossible to know everything about a drug at  the time of approval. Some drug side effects may be difficult to distinguish  from the symptoms of the disease itself and may be sufficiently rare as to go  undetected during the clinical trial process. When health care providers and  their patients look for the best course of treatment, the benefit-to-risk  assessment may vary. Cancer patients undoubtedly desire and deserve the safest  products possible, but within the continuum of cancer progression— from  prevention to late-stage illness&mdash;patients are often willing to accept higher  levels of risk. Cancer is not just one disease, but rather hundreds, all with  different characteristics and affecting different types of people, most of whom  hold hope in an innovative pipeline to develop new products to help their  condition.

Because of the amount of time it currently takes to develop new medicine, the  community became concerned that an overemphasis on safety could result in more  pre-approval requirements for new drugs that would be unlikely to improve a  product’s overall safety profile and also delay patient access, and eventually  reduce the incentive for researchers to develop new therapies for patients most  in need of innovation.

Unfortunately, there’s no quick fix or guarantee for the safety of  prescription drugs, but the appropriate balance of safety and innovation must be  guided by science. Advanced science will yield improved methods for evaluating a  new product’s safety and efficacy while better identifying populations at risk  for adverse effects.

Through significant efforts within Congress, the reauthorization of PDUFA  takes important steps to further incorporate science and technology into the  regulatory arena. Among many updates, this legislation increases the FDA’s  ability to interact with private health care databases to identify risks that  are virtually undetectable in a relatively small pre-approval environment,  without slowing patient access to beneficial new products.

The FDA plays a vital role in the continuum of research as the nexus between  scientific discovery and clinical application. Safety is paramount, but  increased resources for programs like the Critical Path Initiative at the FDA  will help bring innovative discoveries to patients. The public, patients, and  policymakers must recognize the need for ongoing FDA support.

—Ellen V. Sigal, PhD, is the chairperson and founder of Friends of  Cancer Research, a nonprofit organization dedicated to raising awareness and  providing public education on cancer research. The full drug safety report,  “Drug Safety & Drug Efficacy: Two Sides of the Same Coin,” can be found at  

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