The Proper Role of the FDA for the 21st Century

The current medical marketplace is vastly different from the marketplace that existed when the basic elements of the current Food and Drug Administration (FDA) law were passed. Despite rapidly advancing technology and patients’ increasing desire to try new drugs and devices, the FDA has strayed significantly from the statutorily defined safety and effectiveness standards for drug approvals. The FDA now very often demands proof of clinical utility, including survival and disease outcomes, as a requirement for premarket approval. But hard proof of clinical utility is elusive, even for drugs that are clearly shown to be safe and effective as labeled. Rather than blocking safe and effective drugs on such grounds, we should allow physicians and patients to make outcome-oriented decisions, and rely on the medical marketplace to drive physician adoption of safe and effective drugs that prove to have great clinical utility in real-world settings.

The FDA has made significant incursions into private health decisions by exercising its public health mandate in a manner that was not intended by Congress. Rather than allowing doctors and their patients to determine how best to use a drug and ensuring that drugs placed in the market are safe and effective, the FDA has increasingly become concerned with controlling the outcomes of future judgments by physicians and patients regarding benefits and risks. The new medical marketplace should refocus the FDA on safety and effectiveness, leaving patients and doctors to evaluate and determine benefits and risks based on their experiences with the drugs.

There are several steps Congress can take to put the FDA back into its proper role: prohibit consideration of off-label uses, use surrogate endpoints, explicitly define safety, and explicitly define effectiveness.

Click here to read the full publication →