Lessons from Thalidomide

In 1960, Richardson-Merrell – a pharmaceutical company – aimed for quick approval from the FDA for the drug thalidomide to enter the US. The drug was popular in Europe and was touted as a treatment for a variety of ailments including anxiety, sleep issues, nausea, and more. The new drug application was received by Frances Kelsey, a physician and pharmacologist with the FDA. Kelsey resisted the pressure to fast track the drug, citing an insufficient number of controlled studies to establish proper risks. This was especially important because one of thalidomide’s recommended uses was for the treatment of morning sickness. Kelsey requested more data from Richardson-Merrell to show that thalidomide was safe for use during pregnancy.

At the same time that Kelsey requested more information, Estes Kefauver (a senator from TN) was independently conducting an investigation of the US drug industry. As part of his investigation, Kefauver learned about thalidomide’s severe birth defects abroad through the work of Helen B. Taussig, a Johns Hopkins University professor and pediatric cardiologist.

In the 1960s, 10,000 children worldwide were born with phocomelia, a shortening or absence of limbs, as a side effect of the drug Thalidomide.

When studies revealed that about 10,000 children worldwide were born with severe birth defects as a result of pregnant mothers taking thalidomide, the drug application was recalled. Kelsey was celebrated for not allowing thalidomide to be approved for sale in the US. For more on the US Regulatory Response to Thalidomide.

How does this relate to today?

Video URL: https://youtu.be/AonvffI6gng?t=2682 (watch from 44:47 to 46:12)

Certain drugs have been identified as having the potential to treat COVID-19. Compassionate Drug Use is the use of a new or unapproved drug to treat a seriously ill patient when no other treatments are available. For a small subset of people as the last resort, this is acceptable; however, imagine if a COVID-19 virus medication is released for mildly ill patients and it is advertised to help prevent mild cases from becoming more severe? The drug will lack proper research and may have unintended and severe side effects. These poorly studied “treatments” may not be in the best interest of the more than 80% of people who would naturally recover on their own. 

There ARE reliable studies underway.

Interested in Participating in a Clinical Trial?

You can sign up for the Chloroquine/ Hydroxychloroquine Prevention of Coronavirus Disease (COVID-19) in the Healthcare Setting (COPCOV) clinical trial by clicking the link below. The investigators are recruiting 40,000 patients and will use appropriate research methods.

Be well!

Lester Hartman, MD MPH
“Proactive in your child’s care. Empowering families for over 60 years.”

Follow me on Twitter @DrHartmanWMPEDS