By: Margaret Hamburg, M.D. 

Fifty years ago, the vigilance of FDA medical officer Dr. Frances Kelsey prevented a public health tragedy of enormous proportion by ensuring that the sedative thalidomide was never approved in the United States.  As many remember, in the early 1960’s, reports were coming in from around the world of countless women who were giving birth to children with extremely deformed limbs and other severe birth defects.  They had taken thalidomide. Although it was being used in many countries, Dr. Kelsey discovered that it hadn’t even been tested on pregnant animals.

Dr. Kelsey’s reaction to thalidomide exemplifies the FDA’s mission: protecting and promoting the health of the American people, using science for regulatory decision-making.
Now I know that in some circles regulation is viewed as a roadblock to innovation and economic growth. But in actuality, when done right, regulation isn’t a roadblock; it’s the actual pathway to achieving real and lasting innovation.

Smart, science-based regulation instills consumer confidence in products and treatments. It levels the playing field for businesses. It decreases the threat of litigation. It prevents recalls that threaten industry reputation and consumer trust, not to mention levying huge preventable costs on individual companies and entire industries. And it spurs industry to excellence.

The tragedy of thalidomide led to changes that strengthened both the regulatory and scientific environment for medical product development and review.

In response to the public uproar, in 1962 Congress enacted the Kefauver-Harris amendments to the Federal Food, Drug and Cosmetic Act. Thanks to these new amendments, manufacturers had to prove that a drug was not only safe, but also effective. Approvals had to be based on sound science. Companies had to monitor safety reports that emerged postmarket and adhere to good manufacturing practices that would lead to consistently safe products. And there were new protections for patients.

The amendments not only benefited patients, they helped industry, raising scientific standards that eventually ushered in today’s sophisticated, science-based life sciences industry.

For the very first time, many companies put in place research and development programs, including the design and implementation of controlled clinical trials. Major therapeutic breakthroughs resulted, including the use of beta blockers in patients after a heart attack and angiotensin-converting enzyme inhibiters to improve survival in patients with heart failure. All of these were good news for public health and for corporate bottom lines. The best drugs and treatments rose to the top, not simply those that were most heavily marketed.

The Harris-Kefauver Amendments created a culture of quality and innovation that laid the foundation for our current regulatory environment which fosters a domestic pharmaceutical industry that is second to none.

Going forward, smart regulation requires regulatory flexibility that responds to changing situations, new information and new challenges. It also demands that we advance regulatory science: the knowledge and tools necessary for the meaningful and timely review of products for safety, efficacy, quality and performance.

Thalidomide, once again, is a good example. It came back on the U.S. market in 1998 after data showed it was safe and effective to treat a complication of leprosy. In an appropriate balancing of benefit and risk, FDA required strong safety monitoring and a strict dispensing plan before approving the drug.

Regulation such as this requires a strong, robust FDA, one endowed with the necessary resources to ensure smart, sound, science-based regulation.

Margaret Hamburg, M.D., is Commissioner of the U. S. Food and Drug Administration.