Effect Measure


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.


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.


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.


  1. #1 caia
    March 15, 2007

    And of course, in the context of AIDS, rapid approval made sense. Too bad it sounds like they’re using the same process in approving drugs to treat toenail fungus and erectile disfunction.

    How hard would it be to make it so fast approval was only the goal when lives were at stake, and then only when any risks of a new drug appeared to be far lower than the risk of death in untreated patients? So that a drug for stage 4 breast cancer could go through on a fast track if it showed effectiveness in clinical trials, but a drug for insomnia (and I am not dismissing insomnia) could not?

    I hope that’s kind of what “New performance goals must be linked with safety or other public health outcomes, not just speedy approval decisions,” implies.

  2. #2 bar
    March 16, 2007

    Revere: Looks like your editor isn’t compatible either:)

    I didn’t realize just how badly the FDA was set up. Imagine that, FDA will test your drug (thus absolving you of responsibility) if you have the cash to pay them up front (thus eliminating all the snake oil salesmen’s drugs.) In retrospect it is obvious. A bureaucrat (or a lobbyist) had to have designed the system.

    Wouldn’t it have been more sensible to have had a Pharma (drug manufacturer) submit a drug with test results, and request FDA certification? FDA could then analyze procedures and issue a safety rating, (out of 10) based on Pharma’s results. (I see no reason why FDA should judge efficacy, only safety).

    The suggested option might permit snake oil salesmen to enter the market with patent medicines, but so what? If “patent medicine” does no harm, it’s probably ahead of a lot of existing Pharma drugs already. And just maybe it will be found to be good for something.

    If after release of a drug a whole bunch of users had negative symptoms, then the Pharma would be liable for damages depending on repeatability of submitted test results and FDA safety rating. A Big Pharma might end up owing $billions if they cheated the test results. Of course Snake oil salesmen could not be sued for $billions, but I would expect users to take that into account before using the “patent medicine”.

  3. #3 Tim B
    March 16, 2007

    Revere, I just sent you a copy of the cartoon you were looking for. I sent it to the address in your contacts page and the subject says “FDA cartoon”.