Pa-Doofus

You probably don't know what PDUFA is (pronounced pah-doofah) but it's about to expire. Which is good. If they let expire. "They" are our friends in congress. PDUFA stands for the Prescription Drug User-Fee Act and it is an integral part of the FDA drug safety program. You know. The one that brought us Vioxx. I wish I had the great cartoon I saw at the time of the Vioxx trial that showed a bottle of medicine with a label on it that said, "FDA Approved Drug." On the label was a small box that said: "Warning: contains FDA approved drug." So what's PDUFA?

The idea behind PDUFA sounds very reasonable. Big drug companies get a benefit out of the FDA doing all the work necessary to approve their drugs. They should pay for that service. So PDUFA establishes a system of user fees, wherein brand name drug companies pay costs for every new drug of theirs the agency reviews. Sounds great, but it turns out there are unintended consequences, consequences so severe that a group of 22 top drug safety experts has sent an open letter to the lawmakers involved with PDUFA reauthorization asking them not to continue the user fee system.

"User fees may appear to save the taxpayers money, but at an unacceptable cost to public health," the letter warns, citing findings of a panel of experts recently convened by the Institute of Medicine (IOM) to address drug safety at the FDA. Four of the letter's signers served on that IOM panel, and six signers are former top staff of FDA or the Department of Health and Human Services.

[snip]

Unlike other user fee programs in the federal government, PDUFA is negotiated with representatives of fee payers - in this case, the Pharmaceutical Research and Manufacturers of America (PhRMA). Under this arrangement, risk management for drugs already on the market receives little attention, and FDA's ability to conduct post-marketing drug safety surveillance is limited.

[snip]

If PDUFA must be reauthorized to ensure an adequate FDA budget, the signers recommend that it be re-authorized for no more than one year and that it include the following characteristics:

⢠Allow FDA leadership to determine how the agency allocates the fees collected to fulfill all aspects of its mission.
⢠Deadlines or targets for speed of review must be eliminated or modified to allow flexibility and adequate time for evaluation and analysis by reviewers.
⢠New performance goals must be linked with safety or other public health outcomes, not just speedy approval decisions.
⢠Adequate resources must be made available for scientific research and training for FDA scientific and medical staff, including in drug safety epidemiology and risk management.

The letter concludes, "The FDA's mission is to protect and advance the public's health. As it currently exists, and would exist in its proposed form, PDUFA stands in the way of this objective." (news release from the Scientific Knowledge and Public Policy (SKAPP) project).

The whole question of reauthorizing legislation with an acronym with a weird pronunciation must seem like arcane kind of inside-the-Beltway nonsense. But the PDUFA problem goes to the heart of the drug safety system, the need to uncouple assessment of safety from those who profit from drug approval. Unlike the brand-name drugs, it is a generic problem (that's a pharmaceutical joke).

In the last 20 years the drug safety problem has been successfully reframed by the drug companies and their allies in congress as one of cutting time to market. The AIDS community pushed this idea in the special context of getting potentially life saving drugs out faster. In truth, though, most drug approvals don't involve drugs that are the only hope for otherwise doomed patients. Speed of approval has now become an objective in itself and safety has suffered.

In reality, the FDA has being reduced to a paper shuffling agency. Morale among their scientists is low as they become more and more certifiers of information paid for or provided by the drug companies.

PDUFA is a symptom of a deeper problem.