Thalidomide and birth defects

DEFINITION: Sedative drug prescribed for pregnant women in the late 1950s and early 1960s to combat morning sickness until it was found to cause serious physical deformities during fetal development.

SIGNIFICANCE: When taken by pregnant women between the twentieth and thirty-sixth days after fertilization, thalidomide caused severe birth defects. The thalidomide tragedy showed that governments need to be scrupulous in determining the possible risks posed by new drugs before granting permission to manufacturers to market those drugs. Forensic scientists must investigate unusual medical conditions that appear in the population as rapidly as possible so that regulations can be put in place to avert disaster.

Prior testing of thalidomide had been seriously flawed when the drug was introduced into West Germany during the late 1950s and marketed vigorously as an antidote against a variety of human ailments, including morning sickness.  By the late 1950s, thalidomide was widely used in West Germany, where it was , in some cases, available without a prescription. An advertisement by the German company selling the drug misleadingly declared that thalidomide was an antidote for morning sickness, that it relieved tensions associated with pregnancy, that it could be taken as often as necessary, and that it would harm neither pregnant women nor their unborn children.

Discovering Deformities

In December 1961, William McBride, an Australian physician, reported in a major medical journal that he had attended the births of a number of babies who showed severe physical abnormalities (especially, phocomelia, or underdeveloped limbs) and whose mothers had used thalidomide. Shortly thereafter, German physicians reported similar outcomes and presented pictures of newborns with various deformities, including some with finlike appendages attached to their shoulders instead of arms and hands.

At that time, thalidomide was being sold in forty-six countries under different brand names. It has been estimated that over ten thousand newborns throughout the world suffered serious defects as a result of their mothers’ use of thalidomide. The injuries occurred primarily in Western Europe, Canada, Australia, and Japan. In West Germany, after prolonged court hearings,  compensation funds were established through contributions from the manufacturer and the government to provide financial support and pensions to thousands of individuals affected by thalidomide.

In the United States, the drug company Richardson-Merrell applied to the Food and Drug Administration (FDA) in 1960 for approval to market thalidomide. The application went to Dr. Frances Kelsey, who possessed a doctorate in pharmacology and a medical degree but had been working at the FDA for only a month. Kelsey was pressured by her superiors and by the drug company to approve thalidomide, but she insisted that better test results had to be obtained before she would recommend approval. As a result of Kelsey’s stance, only a limited number of cases of thalidomide-related deformity among newborns occurred in the United States. In 1962, Kelsey was awarded the President’s Award for Distinguished Federal Civilian Service for her firm stand against federal approval of thalidomide.

Remedial Actions

The powerful lesson that emerged from the dire consequences of the failure to block the marketing of thalidomide outside the United States was that governments must be vigilant in monitoring new products produced by drug companies and should strongly resist companies’ efforts to enhance their profits by bringing drugs into the marketplace prematurely. In addition, forensic specialists need to analyze the reported results of tests on new drugs carefully to ensure that the testing has been conducted on adequate samples of animals and humans and that the results are accurately presented.

Following the thalidomide disaster, an important step was taken toward protecting the public in the United States with the passage in 1962 of the Kefauver-Harris Amendment, a measure that had been languishing in congressional committees through many sessions. The bill eliminated a previous provision of US law that allowed drug companies to sell new drugs to the public if the FDA did not say otherwise for six months after the drugs were submitted to the FDA for approval. Since the passage of the amendment, drug companies can sell new drugs only after the FDA has affirmed that the products are safe and effective based on “substantial evidence.” Such evidence must include the results of carefully conducted scientific trials using matched groups where possible; that is, during the testing of a new drug, persons in one group undergo treatment with the drug while those in another are given a placebo, a harmless pill that resembles the drug being tested.

In 1998, the FDA approved thalidomide for limited uses. It has been shown to provide dramatically effective treatment for complications of Hansen’s disease (leprosy). Clinical trials for erythema nodosum leprosum (ENL) demonstrated substantial symptom relief in a majority of treated patients, compared with minimal improvement among those receiving a placebo. Subsequently, thalidomide was also approved for use in the form of combination therapy for treating multiple myeloma. Thalidomide has also been approved for use in the treatment of the symptoms of acquired immunodeficiency syndrome (AIDS), some forms of cancer, and various skin conditions.  

In 2023, the government of Australia formally issued a national apology to all citizens affected by the tragedy. The government reopened its Australian Thalidomide Survivors Support Program to ensure that anyone who was affected by thalidomide could secure access to support. Additionally, Australia unveiled a national site of recognition in Canberra for survivors of the thalidomide incident.

In May 2025, the US FDA issued draft guidance for thalidomide oral capsules, detailing bioequivalence and study design requirements for generics and reaffirming strict Risk Evaluation and Mitigation Strategy (REMS) with Elements to Assure Safe Use (ETASU) controls due to severe teratogenic risk.

Bibliography

Albanese, Anthony. “National Apology to Thalidomide Survivors and Their Families.” Australian Department of Health and Aged Care, 29 Nov. 2023, www.health.gov.au/ministers/the-hon-mark-butler-mp/media/national-apology-to-thalidomide-survivors-and-their-families. Accessed 21 Jan. 2026.

Bennett, Chloe. “History of Thalidomide.” News-Medical, 15 Mar. 2023, www.news-medical.net/health/History-of-Thalidomide.aspx. Accessed 21 Jan. 2026.

Brynner, Rock, and Trent Stephens. Dark Remedy: The Impact of Thalidomide and Its Revival as a Vital Medicine. Basic Books, 2001.

Mason, David. Thalidomide: My Fight. Allen and Unwin, 1976.

Miller, Marilyn T., and Kerstin Ström-Land. “Teratogen Update: Thalidomide: A Review, with a Focus on Ocular Findings and New Potential Uses.” Teratology, vol. 60, no. S1, 1999, pp. 306–21, thalidomide.ca/wp-content/uploads/2017/12/article-miller-stromland-teratogen-update.pdf. Accessed 21 Jan. 2026.

Rehman, Waqas, et al. “The Rise, Fall and Subsequent Triumph of Thalidomide: Lessons Learned in Drug Development.” PubMed Central (PMC), 28 Feb. 2013, www.ncbi.nlm.nih.gov/pmc/articles/PMC3573415/. Accessed 21 Jan. 2026.

Roskies, Ethel. Abnormality and Normality: The Mothering of Thalidomide Children. Cornell UP, 1972.

Sjöström, Henning, and Robert Nilsson. Thalidomide and the Power of the Drug Companies. Penguin Books, 1972.

Teff, Harvey, and Colin R. Munro. Thalidomide: The Legal Aftermath. Saxon House, 1976.

United States Food and Drug Administration. Draft Guidance on Thalidomide. U.S. Department of Health and Human Services, Food and Drug Administration, Center for Drug Evaluation and Research, May 2025, www.accessdata.fda.gov/drugsatfda_docs/psg/PSG_020785.pdf. Accessed 21 Jan. 2026.

Winerip, Michael. “The Death and Afterlife of Thalidomide.” The New York Times, 23 Sept. 2013, www.nytimes.com/2013/09/23/booming/the-death-and-afterlife-of-thalidomide.html. Accessed 21 Jan. 2026.