New drugs have the potential to not only extend patients’ lives but also change their quality of life. But are the drugs approved by the FDA reflective of the broader US population? If there were lots of drugs developed for men but not women,or whites but not blacks, health gains from new drugs would be distributed unevenly in the population.
To examine this question, I review FDA’s Drug Trials Snapshots Report for 2019. The document examines the composition of patients enrolled in the clinical trials used to provide evidence for all 2019 drug approvals.
The first thing of note is that clinical trials in 2019 favored treatment for women. In the U.S., 51% of the population is female but 72% of people who participated in trials for FDA-approved drugs in 2019 were women. Unsurprisingly, the treatments approved in 2019 were more likely to target the elderly. Whereas only 16% of the US is aged 65 years or older, 36% of participants in clinical trials met this age threshold.
The racial composition of trials for 2019 FDA-approved drugs largely matches the racial composition of the U.S. Trials had slightly fewer Whites and Blacks, slightly more Asians and basically identical numbers of Hispanics as the US population overall.
These average numbers hide significant heterogeneity across drug trials. Many trials with limited or extensive diversity are created so by design. For instance, prostate cancer trials only enroll men and trials for breast cancer treatments will largely enroll women. Further, some treatments affect specific races more than others. The two trials for approved treatments for sickle cell disease, for instance, had 67% and 91% of their trial participants be African-American. The chart below shows some of this heterogeneity with much variability in racial composition and many trials clustered around a 50/50 split of men and women.
In short, it appears that the composition of the 2019 FDA-approved drugs does a good job of reflecting the racial and gender demographics in the US.