Friday, March 14, 2008

Is the Food and Drug Administration on the verge of a breakdown? According to a recent report from an FDA subcommittee, the answer, apparently, is yes.

The report from the agency’s Subcommittee on Science and Technology, concludes that the FDA “cannot fulfill its mission because its scientific workforce does not have sufficient capacity or capability” — a conclusion that should serve as a wake-up call for all of us. Some have suggested that the agency’s problems stem from its being stretched too thin due to inadequate funding. But more funding alone will not be sufficient to equip the FDA with the resources necessary to fulfill its mission in the 21st century, especially when it comes to approval of new drugs.

Plain and simple, the FDA needs new blood. The agency needs to inject a dose of fresh scientific talent and critical thinking into the drug assessment and approval process.

Consider the FDA’s decision last week to grant “accelerated approval” for Genentech’s blockbuster drug Avastin, previously approved for treating lung and colorectal cancers, for the treatment of breast cancer. Hailed by some as an audacious leap in the FDA’s approval process, it was in reality an overly conservative and tentative step, grounded in a contorted use of the accelerated approval provision, which is intended to allow faster access to promising experimental drugs. Instead, more trials are now required, with more time and more money required to prove a survival advantage that may prove elusive.

Almost three years ago, Avastin was shown in a major study to significantly prolong the time patients with breast cancer remain in remission. Most scientific experts consider this benefit “progression-free survival,” sufficient for full approval, and many oncologists have been using Avastin as an “off-label” drug for this purpose.

The National Institutes of Health (NIH) funded the breast-cancer study through a consortium of clinical trial sites. Many of the country’s leading academic and private institutions participated. Findings were presented as late-breaking news at the 2005 Annual Meeting of the American Society of Clinical Oncology, and results were published in the New England Journal of Medicine in December 2007.

But the FDA ignored these experts. When the clinical trial data were evaluated by FDA’s cancer-drug advisory committee in December, only two of the panel’s nine members were breast-cancer experts. The panel’s chairperson, an expert in prostate cancer with little apparent expertise in breast cancer, cast the deciding vote against approval.

The FDA needs to keep pace with current thinking in the broader scientific community and avoid needless additional trials like those now awaiting Avastin. We need experts in various medical fields to work with the FDA on both the design and review of clinical trials. Providing research funding or other incentives to leading academicians and industry scientists is one way to get these experts involved.

Outside academic experts who participate in competitively funded FDA programs should receive professional honors equal to those given to winners of other prestigious grant awards, as is the case with peer-reviewed NIH grants. Similar steps could be taken by the FDA with industry scientists, who could be welcomed as FDA research partners and, in turn, rewarded by their companies for pursuing the opportunity to gain valuable experience.

The FDA should also utilize existing federally funded cooperative clinical trial groups — collections of academic and private medical centers — to design and conduct clinical studies. And, unlike the case with the Avastin study completed by a prestigious NIH-funded consortium, the FDA should confidently rely on their results. The FDA could also establish regulatory science centers of excellence on the campuses of universities, similar to the idea of embedding national research laboratories within universities.

An insular FDA cannot maintain the level of scientific talent required to regulate and facilitate new product development in an era of unprecedented and unpredictable scientific advancement. But reaching out for a healthy dose of fresh thinking from academia and the private sector may be just what the doctor ordered.

Dr. Richard Miller is president and CEO of Pharmacyclics and adjunct professor of oncology at Stanford University Medical Center.

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