Transforming Clinical Trials for Women in 2024

By Juan Camilo Arjona Ferreira, chief medical officer & head of R&D, Organon

Despite making up over half of the world's population, women only made up, at most, 34% of participants in Phase 1 clinical trials for new drugs approved by the FDA CDER between 2013 and 2015. Although the establishment of the NIH inclusion policy requiring federally funded trials to include female participants and assessing gender-specific treatment effects 21 years ago was a significant step forward for women's health, low female participation persists in certain therapeutic areas. This perpetuates gender biases in drug development and widens our knowledge gaps on how drugs affect women, which may negatively impact their health outcomes.

As pointed out in research by McKinsey & Company, relying on male physiology for healthcare solutions has resulted in suboptimal outcomes for women. Between 1997 and 2000, eight out of 10 prescription drugs withdrawn from the market posed greater health risks for women than men. And, women have been diagnosed significantly later than men across more than 700 diseases.

Change is long overdue, and it begins with a fundamental shift in how we design clinical trials, placing women's perspectives and needs at the core of planning and implementation. At Organon, we have been challenging the status quo in women's health since our inception. We have explored innovative approaches to transform clinical trial practices, enhancing diversity, and prioritizing health equity.

Here are five opportunities we have identified to bridge the current data gaps and better serve women:

1. Leverage technology to reduce barriers to participation. There are many barriers that prevent women from participating in clinical trials — from transportation issues to a lack of childcare to the need for paid leave — and these barriers disproportionately affect communities of color. Further, women-specific conditions such as endometriosis can be debilitating, emphasizing the need to minimize clinical trial travel time and frequency. For example, by implementing an app on participants’ personal devices during our ELENA trial for endometriosis, Organon captured data around efficacy, quality of life, and safety during clinical development. This use of technology encouraged digital participation in the trial and allowed us to help reduce accessibility, transportation, and paid leave barriers.

2. Integrate women’s voices into trial design. Listening to women and incorporating their insights into how we design clinical trials is key to developing more effective and patient-centric research. At Organon, we capture women’s unique experiences and ideas to ensure that the science we pursue will make a meaningful impact in their lives — and we’re constantly looking for new ways to embed insights from women into our work. It’s this commitment that led us to partner with Oracle Life Sciences to conduct listening workshops that helped to inform the design of our ELENA trial. Our workshops involved patients writing heartfelt letters to their disease (in this case, endometriosis), mapping the timelines of their journey from onset to diagnosis and journaling their experiences. By integrating the insights we gleaned into our study protocol, we were able to address barriers, enhance participant comfort, and foster a strong rapport between the researchers and the women involved.

3. Use data to reduce racial disparities. Using data-driven site selection to identify locations where specific patient populations are concentrated improves accessibility for patients, thereby helping to ensure individuals from diverse backgrounds have equal opportunities to participate. The alarming reality in the United States that Black women are three times more likely to die from pregnancy-related causes than white women highlights the urgent need for change. By reaching out to patients in the communities where they live and gathering more data from underserved and underrepresented populations, we can develop culturally relevant practices that address the historical mistrust of the healthcare system prevalent in marginalized communities and promote inclusive and equitable healthcare outcomes. For instance, programs focused on increasing transparency, improving communication and creating more welcoming environments can help address mistrust.

4. Create new protocols for integrating pregnant women into clinical trials. While industry has made inroads in recent years when it comes to more balanced racial and ethnic representation in clinical trials, pregnant women have historically been underrepresented. This contributes to substantial blind spots in our understanding of how certain treatments may impact expectant mothers and their babies. One important avenue to consider is implementing stepwise trial protocols that gradually integrate pregnant women into clinical trials. This means trials would begin with people who are not pregnant or lactating, and then expand to include people who are, focusing on women who require specific medical treatment for their own health, after a certain baseline of safety has been established. An example of the success of this approach can be seen in the inclusion of pregnant women in early studies of antiretroviral therapy for HIV, which was driven by the knowledge that the lifelong risk of mortality among infants infected by their mothers could be prevented by treatment during pregnancy.

5. Invest in new approaches to improve our understanding of how existing drugs impact women. Policymakers, regulators, and manufacturers must invest in approaches to improve real-world data (RWD) collection to improve our understanding of how existing drugs impact pregnant and lactating women. For example, this could include implementing enhanced data repositories showing how existing drugs impact pregnant and lactating women and analyzing and sharing the data as permitted with other manufacturers, researchers, and HCPs.

Although there is still much work ahead to achieve health equity, the tide is turning with greater attention and investment in women's health, including the White House's initiative to propel women's health research forward. We now have the tools and knowledge needed to transform the health of women everywhere, but it demands collaboration and action from stakeholders across sectors. Let's seize this opportunity and make this year the most transformative yet for women's health.

About The Author:

As Organon's Head of Research and Development and Chief Medical Officer, Juan Camilo Arjona Ferreira, MD, leads the advancement of a diverse portfolio of medicines and solutions that focus on improving the health of women and all patients. In this role, Juan Camilo contributes his strategic and dynamic perspective, overseeing and strengthening the organization’s scientific, medical and regulatory capabilities in support of its long-term mission.

Juan Camilo has over 20 years of experience in clinical research and development. Before joining Organon, he was the Chief Medical Officer of Myovant Sciences Inc., where he provided strategic direction and execution oversight to the global development and medical affairs teams. Previously, he was SVP of clinical development at Shionogi Inc., where he led the development of clinical strategy and provided execution oversight for late-stage development programs. Prior to Shionogi, Juan Camilo was the Executive Director of Clinical Research in Women’s Health at Merck and led the product development teams for all programs in contraception and women’s health. Earlier in his career, Juan Camilo practiced as an OB-GYN in university hospitals and in private practice.

Juan Camilo received his M.D. and post-graduate specialist training in obstetrics and gynecology at Colegio Mayor del Rosario in Bogota, Colombia.