International Women’s Month: The Woman Who Saved America from a Pharmacologic Disaster
In the 1950s, a new sedative called thalidomide, initially advertised as a sedative that would allow users to undergo a deep sleep in the absence of a hangover, was widely prescribed to pregnant women in Europe as a treatment for pregnancy-related nausea. At the time, basic testing was done on the drug, and it was considered not to have any toxic effects on humans. However, unlike today’s level of rigorous testing, the drug was not analyzed for any potentially dangerous effects on fetal development.
By the 1960s, the effects of the wonder drug were clear: thalidomide caused nerve damage in the hands and feet of adults. When taken in early pregnancy, it causes impairments such as limb difference, sight loss, hearing loss, facial paralysis, and impact on internal organs. Even one dose was enough to cause significant impairments, with congenital disabilities such as very short or missing arms and legs, missing parts of the ears, and deafness appearing in nearly 80% of babies born to mothers who had taken the drug.
Despite the high incidence of birth defects in Europe and across the globe, the US was spared from the horrors of thalidomide by the efforts of one woman. Frances Oldham Kelsey earned a PhD in pharmacology in 1942 and her MD in 1950, both from the University of Chicago. It is worth noting that the department head at the University of Chicago was unaware of common spelling conventions and assumed that Frances was a man when he decided to admit her to the program. Her acceptance letter was addressed to “Mr. Oldham.”
In 1960, Dr. Kelsey was a Food and Drug Administration reviewer when the application to begin mass marketing thalidomide in the US landed on her desk. As a reviewer of New Drug Applications (NDA), she was one of three people charged with determining a drug’s safety before it could be made available for public consumption. After a thorough review, she rejected the application for thalidomide because it lacked sufficient evidence of safety through rigorous clinical trials. She found the clinical trials insufficient and reliant on anecdotes and physician testimonials rather than sound scientific study.
Her decision was met with resistance. Richardson-Merrell, a company based in Ohio, had submitted the NDA to the FDA, assuming it would be approved. The company had a launch date 6 months out and was also distributing samples while touting the sedative as safe and effective against nausea in early pregnancy. Richardson-Merrell submitted additional data, which was inadequate; again, Kelsey rejected it. This cycle would repeat, resulting in company officials making more than 50 in-person visits to the FDA’s offices to campaign for the drug’s approval. After all, the company had more than 10 million tablets ready for distribution, and executives were looking forward to their year-end bonuses. On March 8, 1962, Richardson-Merrell withdrew its application from the FDA after being confronted with the seriousness of the drug’s side effects.
Kelsey’s refusal to approve thalidomide for sale in the US prevented the births of thousands of seriously malformed babies. In August of 1962, President John F. Kennedy awarded her the President’s Medal for Distinguished Federal Civilian Service for her exceptional judgment in evaluating the drug. Dr. Kelsey was only the second woman to receive this award—the highest honor that can be bestowed upon a US civilian. Her story is one of perseverance, dedication, and an unwavering devotion to science and the scientific method. It is also a reminder that the right thing may not be the popular thing, the profitable thing, or the easy thing. But there is no right way to do the wrong thing.