Prairie Doc Perspective the week of October 15th, 2023
By Debra Johnston, MD
Aristotle is said to have referred to the female as a mutilated male, and this philosophy seems to have carried forward into much more modern times.
In 1977, official FDA guidelines recommended that women of “childbearing potential” be excluded from early stage clinical trials. Men, particularly white men, were considered the ideal model, from which the success and side effects of any particular treatment could be judged. Somehow, women’s menstrual cycles made them too difficult to study, while simultaneously being irrelevant to the results. The thalidomide tragedy quite reasonably brought fetal wellbeing to the front of researchers’ minds, but there was reluctance to wait for animal teratogenicity data before starting human trials. Companies often interpreted childbearing potential to mean childbearing age, and then extended that ban to all women, in all stages of research. Studies carried out in the mid to later part of the last century frequently enrolled thousands of participants, but no women at all. This was even true in purely observational studies, where nothing was being trialed or tested.
Then, in 1993 congress passed a mandate that women be included in NIH funded trials, and the FDA changed their guidelines. Now they said that the population studied in trials should be representative of the people likely to be treated with an intervention if it were approved. This meant that most studies had to include people of different ages, races, and genders.
Once scientists started looking, it became evident that the differences among us translate to differences in the way diseases develop, the way conditions present, and the way we respond to potential treatments. We may need different doses. We may experience different side effects. We may need different treatments altogether. This can be true for differences based on age, race, health status, and, of course, sex.
The scientific landscape is very different today than it was in 1993, but we still have a long way to go. A recent review of studies involving cardiovascular disease, cancer, and mental health interventions found that just over 40% of study participants were women. Cardiovascular disease and cancer affect men and women almost equally. In mental health research the situation is worse: women make up approximately 60% of those affected, but still, just over 40% of those enrolled in trials.
Everyone deserves to know that the recommendations their doctor makes are backed by science that includes people like them. Many treatments work just as well in people of different races, ages, or genders. But many don’t, and we won’t know if we don’t ask that question.