Following the president’s recent executive order dismantling diversity, equity, and inclusion policies, the main page for the FDA’s Diversity Action Plan guidelines — requiring pharmaceutical companies to test drugs on diverse populations before approval — has been down since Jan. 23.
These guidelines were established under the Food and Drug Omnibus Reform Act of 2022, enacted by Congress to promote inclusivity in clinical trials.
It’s upsetting to see. But I predict those mandates will eventually come back — because we need them.
I have sat on more than 20 drug advisory committees, which are groups made up of experts that offer guidance to the FDA on a range of topics. From 2017 to 2024, I was the consumer representative on the FDA’s Drug Safety and Risk Management Advisory Committee, requiring me to represent the general public’s priorities and concerns during the development of new drugs.
During advisory meetings, the group is tasked with reviewing and commenting on the data that the FDA uses to determine drug approval, and I can tell you firsthand — the trials we looked at were overwhelmingly populated by white men.
At first, I hardly noticed it. Then, I dismissed it. The differences in efficacy and harm between men and women, white men and Black … they would be small, or nonexistent, I hoped.
I was wrong.
Consider this scenario: A 75-year-old woman and her husband, age 76, are coincidentally and simultaneously diagnosed with early-stage Alzheimer’s disease. Their doctor places them both on Leqembi, recently approved by the FDA for use in men and women with early-stage Alzheimer’s. The husband has a positive cognitive response to Leqembi, but his wife does not. Her Alzheimer’s progresses, and she loses touch with reality, her children, her life. Her husband is still able to socialize and play golf.
Why? We don’t entirely know. Women were not analyzed separately from men in the clinical trials for Leqembi. It was only when researchers examined subgroup data that they realized a stark difference: 43% of men responded to the drug, compared with just 12% of women. Because the trials were not required to assess sex-based differences in effectiveness, the study lacked enough participants to determine whether this disparity is statistically significant. Still, the findings serve as a strong indicator of a potential gap in treatment efficacy.
So now, the children in this scenario realize their mother was exposed to Leqembi’s severe adverse event risk without a counterbalancing likelihood of real benefit. Other drugs, other therapies might have served her better.
This is a hypothetical scenario, but it’s based on a very real issue with a very real drug: Since effectiveness data was initially buried in the supplemental analysis of the original study, the difference in response between men and women remained unknown until researchers went back and unearthed it.
Leqembi’s limited clinical trial is not an isolated incident. Clinical drug trials have historically been built on a narrow foundation. Scientists have long studied white men and applied the results to men of all colors and, likely, titrated the drug down for women’s smaller bodies.
And now, despite decades of awareness, reforms, and new guidelines, marginalized groups — women (including pregnant women), Black people, older adults, and entire ethnic communities — are still being left out of research that directly affects their health.
In fact, the Center for Drug Evaluation Research found that 75% of the participants in the clinical trials for the 53 drugs the FDA approved in 2020 were white. The likely result? New medications are brought to market that work well for white patients but may fail to address the unique needs of others.
Modern medicine is a miracle. But it can’t be a miracle that works for only part of the population and leaves the balance in the dark. We need our drugs evaluated by subgroups — we need diversity in clinical trials. Or we are just guessing about medical care.
Suzanne B. Robotti is the founder of the MedShadow Foundation, executive director of DES Action, and served on the FDA Drug Safety and Risk Management Advisory Committee from 2017 to 2024.