Preventing Public Health Disasters

Earlier this week, FDA Commissioner Margaret Hamburg presented Frances Kelsey with the first in what will be a series of awards bearing Kelsey's name. Fifty years ago, as a new medical officer with the FDA, Kelsey refused to approve US sale of Kevadon, a drug widely recognized by its generic name, thalidomide. The drug was widely used as a sleep aid and anti-morning-sickness drug in Europe, but Kelsey questioned its safety.

While thousands of children were being born in Europe with missing limbs and other thalidomide-induced birth defects, the US avoided such widespread tragedy. In 1962, President John F. Kennedy presented Kelsey with the Distinguished Civilian Service Medal. The Washington Post's Lyndsey Layton explores some of the far-reaching effects of Kelsey's actions and thalidomide's horrific effects:

The thalidomide case transformed the way Americans think about medicine, said Morton Mintz, a former Washington Post reporter who broke the story of Kelsey and thalidomide in 1962. Images of deformed babies underscored the dangers of drugs, and the need for careful scientific vetting by regulators, he said.

"Until that point, people used to think that science, technology and the drug industry could bring only progress," Mintz said. "People used to think new is necessarily better. But Kelsey nurtured a healthy skepticism in the public and the news media."

The United States was not entirely spared in the thalidomide tragedy. Merrell gave the drug to more than 1,000 U.S. physicians to distribute to 20,000 patients as part of an "investigational" trial. In many cases, the patients were not informed they were participating in an experiment. An estimated 40 babies in the the United States were born with deformities as a result.

The scandal prompted Congress to amend the drug law in 1962 to impose strict limits on the testing and distribution of new medication. The regulations required that drugs had to be proved safe and effective, and that patients had to give informed consent to participate in clinical trials. It also required that the manufacturer report any adverse reactions to the FDA.

After the law was changed, the FDA created a division of new drugs and made Kelsey the director of scientific investigations. Her principal responsibility for the rest of her career in the agency was directing drug testing.

The New York Times' Gardiner Harris highlights an important piece of the story: Kelsey's supervisors supported her in the face of complaints from the drug's manufacturer, the William S. Merrell Company. This supportiveness stands in contrast to what Harris described in a 2007 story about a spate of cases in which FDA safety reviewers were "punished or discouraged" after bringing drug dangers to their supervisors' attention. In his more recent article, Harris notes that Hamburg's honoring of Kelsey may be a signal about a shift at FDA:

The inauguration of the Kelsey award may also be a telling sign of where Dr. Hamburg stands in a series of internal agency struggles. For much of the past two decades, the F.D.A. has emphasized speed over certainty in its decisions -- an industry-friendly stance that plays down safety concerns in favor of getting potential cures to the market as swiftly as possible.

But a series of drug, medical-device and food-safety controversies have led some agency medical officers to insist on better information before approving products and to lobby internally for risky products to be pulled from the market, putting the speed-oriented old guard on the defensive. A celebration of Dr. Kelsey, the patron saint of the agency's safety-first faction, is bound to cheer those calling for greater caution.

The story of Frances Kelsey and thalidomide reminds us why it's so important to have a strong regulatory system staffed by thoughtful, committed people.