How Well the FDA Works (or Doesn't)

Robert W. Griffith, MD

Americans are unsure about drug safety and the Food and Drug Administration's (FDA's) record in protecting them. Recent controversies about the long-term safety of Vioxx®, drug-coated stents, and the glitazone antidiabetic drugs Actos® and Avandia®, have increased the doubts. A survey has shown that while 82% of those polled trust the FDA when it comes to overseeing prescription drugs, 47% rated its performance as fair or poor. People are concerned about the increasing number of drug-related adverse effects; as many as 3 out of 4 believe that drug safety is more important than speedy new drug approval.

The FDA reviews new drugs based on the results of what are considered the gold standard of drug testing - randomized clinical trials, or RTCs. These employ many (often thousands) of patients treated for long periods (often 1 or 2 years), and provide convincing proof of a new drug's effectiveness, or efficacy. However, the number of patients in the clinical trials and the duration of treatment are not necessarily large enough to detect relatively rare adverse events. Moreover, RCTs are conducted under experimental conditions, with rigid protocols for patient selection and treatments. Real-world conditions are different and potentially more relevant, as a broader set of patients may receive a wider range of treatments. It's clear that some adverse events will only become apparent when the drug is used by many thousands of people in the community.

The FDA and the pharmaceutical companies don't, at present, have the ability to conduct such investigations. Methodology for the collection of real-world data needs to be developed and tested in pilot trials. All this will take years and a lot of funding. Before any new regulations in this direction are implemented, we should be sure that we don't throw the baby out with the bathwater. New drugs bring new benefits (in most cases), and it should be possible to monitor their use during the first few years to be sure of picking up unexpected changes in patients taking them - obvious or far-from-obvious adverse events. This discipline is called "postmarketing surveillance", and it should become part of the FDA's and drug company's duties as soon as a new drug is approved. Problem is, an effective methodology doesn't exist, yet...

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HealthandAge Blog

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