What About Clinical Trial Equity for … Women?

The FDA’s new draft guidance, Diversity Action Plans to Improve Enrollment of Participants from Underrepresented Populations in Clinical Studies, addresses an important opportunity. The recruiting of clinical trial participants who more precisely mirror a disease’s patient population can be a powerful tool to improve overall data sensitivity and, ultimately, more targeted and positive patient outcomes. But the agency’s efforts have to be more than a gesture – and it’s a lot more than just about communities of color.

Diversity in clinical trials is a bell weather issue. We are all learning the nuanced differences between “equality” and “equity.” Equality means that each patient is given the same access to available opportunities. Equity recognizes that each patient has different circumstances and provides the needed resources to reach an equal outcomes. But clinical trial diversity isn’t only a color issue, it’s a gender one as well.

The FDA must prioritize women’s health. One way to start is to insist that female representation in clinical trials be representative of the disease state—not that every trial needs to be 50% female, but that the clinical trial make-up should represent what the actual patient population looks like.

Of course, the same is true for other subpopulations such as communities of color. For example, African Americans overrepresent in cardiovascular diseases. So, saying that 13.6% of cardiovascular disease clinical trial participants should be African American is a false metric. It should be higher.

Women’s health mustn’t be ghettoized in the uterus. Beyond reproductive and “below the waist” issues, we have mostly ignored conditions that impact women either in the majority or significantly. For example, up until very recently, the medical community (aka “male doctors”) did not even think fibromyalgia was a “real” disease, but rather all in the mind of the “gentler sex.”

You want to talk about an equity gap, then first consider the gender chasm:

• Seventy-eight percent of Americans with autoimmune disease are women.
• A woman’s risk of developing Alzheimer’s disease is almost two times that of a man (and not just because women live longer).
• Only 12% ($2.9 billion) of the National Institutes of Health budget is earmarked for women’s focused research.

As a point of reference, over 60 million women (44% of all women) in the USA are living with some form of heart disease. In 2021, cardiovascular issues were responsible for the deaths of 310,661 women, or about one in every five female deaths. For men, heart disease killed 384,886 men in 2021, or about one in every four male deaths. Cardiovascular disease is the number one killer of women in the USA, yet only one third of the participants in cardiovascular clinical trials are female. Why aren’t cardiovascular disease clinical trial participants 50% female? Why aren’t women properly represented in other disease-state clinical trials such as Alzheimer’s disease?

One big exception proves the rule—breast cancer was the first “women’s disease” to come into focus once we began to have a better understanding of the human genome. The drug trastuzumab was the first genetically targeted therapy for breast cancer (HER2 gene) and made it unambiguously clear that not only are women not physiologically identical to men, but that women are not genetically identical to each other.

But breast cancer is not the number one cause of female cancer deaths. Lung cancer is, and non-smoking women are more than two times as likely as non-smoking men to get it.

The FDA should insist that clinical trials look like the disease impacted population. That’s smart regulatory science and smart healthcare policy. Making a clinical trial “look like America” is a political statement and isn’t helpful. And we mustn’t stop with clinical trial participation. We need more women clinical trial designers, principal investigators, and FDA advisory committee members, more women venture capitalists, and more women legislators.

If potty parity is important, policy parity is urgent. What is it about 52% of the population that the healthcare ecosystem doesn’t get?