EXOME

all the information, none of the junk | biotech • healthcare • life sciences

Dr. Frances Kelsey: American Hero, Government Regulator

Opinion

Xconomy National — 

Dr. Frances Kelsey passed away last week at the ripe old age of 101. She was a true hero in every sense of the word. How did she achieve this status? She did not pull someone out of a burning building. Didn’t dive into a lake to rescue a drowning child. Never threw herself on a hand grenade to save troops in combat, although she did find herself on a different type of battlefield. She became a hero in a most un-extraordinary way: she simply did her job in the most thorough, competent, and professional way imaginable.

In the early 1960s Kelsey was a new employee with the FDA in Washington D.C. She was tasked with reviewing an application to approve a new drug, Kevadon, which was to be used as both a sleeping pill and a morning sickness treatment. The drug, which had been approved in Europe some four years earlier, was already earning lots of money for its West German manufacturer Chemie Grünenthal. It was available in West Germany as an over-the-counter drug (sold under the name Contergan), meaning no prescription was required. The William S. Merrell drug company had licensed the drug to sell in the U.S., and had already distributed samples of it to 1,200 doctors.

In those days, drug companies could distribute experimental therapies pre-approval for “testing” by doctors. That primed the pump so everything was in place to sell the drug once approved by regulators. William S. Merrell officials repeatedly complained both to Kelsey and her superiors at the FDA that she was taking too much time reviewing the company’s application, asking too many questions, and costing them millions of dollars in lost drug sales. Powerful pharma lobbyists likely railed to members of Congress about her handling of the application. Senator Estes Kefauver once described them by saying, “These drug fellows pay for a lobby that make the steel boys look like popcorn vendors.” The pressure on Kelsey to approve the application must have been tremendous.

Kelsey, however, did what good scientists are trained to do. She looked carefully at the data, found the safety information unconvincing, and asked for more of it. Her superiors at the agency backed her up. Eventually, information started to come in from Europe that the drug was associated with a specific type of birth defect known as phocomelia. Some children born after their mothers took Kevadon (more widely known these days by its generic name, thalidomide) were born with severe birth defects, including seal-like appendages in place of arms or legs. Some 10,000 children were born in Europe with phocomelia, and it’s estimated that 40 to 50 percent of them died. A much smaller number of children with phocomelia were born in the U.S. as a result of doctors giving samples to pregnant women prior to its approval. Chemie Grünenthal pulled the drug off the West German market in late 1961, although Merrell waited until March of 1962 to withdraw its FDA application.

By carefully considering the application and holding up its approval, Kelsey (and her colleagues at the FDA) prevented what would have been a public health disaster in the U.S. Publication of the thalidomide story by the Washington Post made her (and them) national heroes. For her efforts, John F. Kennedy gave Kelsey the President’s Award for Distinguished Federal Civilian Service in 1962. Public opinion, stoked by the news of the thalidomide disaster, helped facilitate the passage in 1962 of the Kefauver-Harris Amendment to the Federal Food, Drug, and Cosmetic Act of 1938. The Amendment is considered to be one of the best examples of consumer protection legislation, even though its opponents complained that it would “unnecessarily expand the power of government, threatening the viability of the pharmaceutical industry, and inserting Washington bureaucrats between patients and their doctors.”

In 2010 the FDA named the Drug Safety Excellence Award after Kelsey, who fittingly was its first recipient. She continued to work at the agency until her retirement at age 90.

These days, of course, the story would have been written, at least in some quarters, with a completely different spin. We’d be reading about a “petty Washington bureaucrat” who was unnecessarily blocking the release of a marvelous new drug that was desperately needed by the American people. We’d hear how the company that wanted to sell the drug would certainly not have asked that its new medicine be approved without having data showing that it was both safe and effective. Some non-scientist would claim there was doubt about the basic science, and that there were genuine disagreements as to what the data meant. If the science simply hasn’t been decided yet, why hold up the application? Dr. Kelsey’s competency and personal integrity would be questioned. We’d hear how her actions were both an affront to personal freedom and harming the reputation and financial status of Merrell. After all, the drug had already been approved for sale in Europe. Why were regulators unnecessarily delaying its approval here in the U.S.?

Thankfully, that’s not the way the narrative was cast back in the day for the American people. The truth came out, a public health disaster was averted, and important drug regulations were put in place that are still in effect today. Keep this story in mind the next time you hear someone tell you that America can be made great again if we simply remove all of those strangling regulatory hurdles imposed on us by the government.