Women’s History Month: Clinical & Regulatory Perspectives in Women’s Health 

In recognition of Women’s History Month, we spoke with two leaders who bring complementary perspectives to women’s health development: Kathleen Lemaitre, MD, FACOG, Executive Director in Medical Affairs and board-certified OB/GYN, and Stacey Ayres, PhD, Vice President, Regulatory Strategy.

Together, they reflect on how women’s health research has evolved — and what it will take to drive meaningful change over the next decade.

Kathleen, what initially drew you to women’s health, and how has that shaped the way you approach clinical development today?

Kathleen:

What drew me to OB/GYN was the breadth of both disease and lifespan—pregnancy, fertility, endometriosis, gyn cancer, complex benign gyn surgery, menopause—too many to name. And OB/GYNs care for women across the lifespan, managing disease and wellness during adolescence, adulthood, pregnancy, and menopause, frequently serving as a woman’s primary physician for decades. We often have the privilege of treating multiple generations in a family so it’s not uncommon to be seeing a woman, her daughter, and her granddaughter concurrently. As you may imagine, this is extremely gratifying for both patients and physicians alike.

Longitudinal relationships also change how one thinks about disease. As OB/GYNs, we see more than a diagnosis; we see how a given condition or obstacle affects a woman’s education, career, family planning, mental health, and long-term wellbeing.

When I support sponsors developing therapies for preeclampsia, endometriosis, bladder disorders, or dysmenorrhea, I’m thinking about real patients I’ve treated, and I bring that perspective to the conversation and to our proposed strategy for the program. Patient populations with a given condition are tremendously heterogeneous: a 19-year-old with dysmenorrhea and a 45-year-old who has had three C-sections may share a diagnosis, but their clinical realities are very different and the treatments that are most appropriate for one may differ significantly from those which are safest and best suited for the other. That clinical experience helps ensure development programs reflect the patients they’re ultimately meant to serve.

What unique role can a CRO play in strengthening the scientific rigor and execution of women’s health studies?

Kathleen:

Women’s health trials are often more complex than they appear on paper.

First, the population is highly variable — age, hormonal status, reproductive intent, comorbidities, BMI, and cultural differences. Even timing within a menstrual cycle can influence outcomes. That level of heterogeneity demands thoughtful protocol design.

Second, some indications — especially obstetrics — introduce operational challenges that are very different from traditional outpatient studies. Patients present 24/7. Site staff require training across shifts. Enrollment pathways are unpredictable. There is a second “patient “ to consider and women and their partners are often very reluctant to risk fetal exposure in a study setting. These conversations and informed consent require significantly more time, thought, and expertise to navigate responsibly and successfully.

A CRO can add value by bringing real-world clinical and operational insight into early planning:

• Engaging multiple KOLs across regions
• Testing assumptions about enrollment feasibility
• Evaluating whether endpoints reflect patient priorities
• Identifying operational stress points before they become problems

We’ve seen sponsors respond strongly when that broader input is integrated early. It strengthens both scientific credibility and execution.

Stacey, over the course of your regulatory career, what lessons stand out from the women’s health programs you’ve supported?

Stacey:

One key lesson is that regulatory success alone isn’t enough.

You can design a program that satisfies regulatory requirements — appropriate endpoints, acceptable safety framework — but still miss what matters most to women living with the condition.

In many women’s health programs, especially those leveraging 505(b)(2) pathways, the opportunity is to improve existing therapies. But improvement has to be defined through the patient lens. Are we addressing symptom variability? Tolerability? Real-world usability?

Another lesson is that women’s health often intersects with broader biological differences. Increasingly, we’re seeing recognition that sex differences matter across cardiovascular, neurological, and other therapeutic areas — not just reproductive health.

That evolution in thinking is encouraging, but it requires continued alignment between science, regulatory strategy, and patient voice.

What common pitfalls could sponsors mitigate earlier in planning?

Stacey:

Endpoint selection is a common inflection point. If endpoints are chosen without sufficient clinical and patient input, sponsors may find themselves with an approvable product that doesn’t fully meet patient expectations. Ensuring that women’s voices are integral to the development and evaluation of products aimed at treating women’s health conditions is essential for achieving meaningful innovation. It is not enough for clinical programs to focus solely on regulatory endpoints. Studies must also capture the outcomes, experience, and priorities that matter most to women.

Another pitfall is underestimating population variability. The number of variables that shape treatment outcomes in women is often underestimated, yet these factors are vital to fully understanding safety, efficacy, and benefit-risk. Reproductive stage, hormonal cycles, fertility considerations — these aren’t minor details. They can affect inclusion criteria, safety monitoring, and even labeling strategy. Acknowledging this complexity is not only a scientific imperative, it reflects a commitment to delivering therapies that truly meet women’s needs.

Early engagement — with regulators, clinicians, and patient-informed KOLs — can prevent costly failures or course corrections in development programs.

Now let’s hear from both of you. Where have you seen the most meaningful progress in women’s health research over the past decade?

Kathleen:

There’s been a noticeable shift in visibility. Conditions like endometriosis, hyperemesis gravidarum, and menopause-related disorders are finally receiving serious attention.

In the past, many of these conditions were minimized or dismissed. Today, we’re seeing something different — not just advocacy, but patients identifying openly with their experience. At a recent scientific conference, I met several women and their partners who referred to themselves as “survivors” of hyperemesis gravidarum, a term historically used in the setting of sexual assault or cancer That language is powerful. It reflects how serious and life-altering the condition can be — and how long it went under-recognized. The fact that patients feel empowered to describe themselves that way signals a broader shift from silence to acknowledgment, and aptly reflects the deep trauma and lasting sequelae for them and their families.

We’re also seeing improved biomarker development, more targeted research, and greater scientific investment across a wide array of women’s health diagnoses, those that effect women exclusively, differently, or disproportionately. That combination — visibility, advocacy, and data — represents meaningful progress.

Stacey:

We’re also seeing an increased recognition of sex-based differences across therapeutic areas, with conditions frequently presenting and progressing differently in women and men. Cardiovascular disease, for example, illustrates how sex-based differences critically shape disease presentation and outcomes. Women often present later with atypical symptoms, receive fewer invasive interventions, and experience higher complication rates partly driven by hormonal and microvascular differences. These distinctions have important implications for clinical development, necessitating study designs that appropriately account for sex-specific patterns of disease and treatment response to ensure robust, generalizable evidence.

Looking at disease through a biological and gender-informed perspective — not just in reproductive health, but across therapeutic areas — represents real advancement.

Where do meaningful gaps still remain?

Kathleen:

Sustained attention and investment remain challenges. Women’s Health shouldn’t be viewed as niche. It intersects with nearly every family, every community, every generation. In sheer numbers, nearly everyone is a woman or has a woman they are closely tied to—mother, daughter, sister, niece, friend.

Stacey:

Alignment is another gap. Regulatory pathways evolve, but they must keep pace with the rapid scientific innovation now reshaping women’s health.

The FDA’s increasing focus on sex-based differences and its updated Women’s Health Research Roadmap, which prioritizes improving clinical study design, advancing biomarker science, and integrating women-specific data into regulatory decisions, reflects a growing recognition of the distinct needs of women across therapeutic areas. The FDA has also issued multiple guidance documents over the past decade addressing sex-specific data analysis, the inclusion of pregnant and lactating people, and the evaluation of sex differences in device trials. That evolution reflects recognition of prior gaps and an effort to strengthen inclusivity in research. As scientific understanding of sex-based biology and life-stage variability continues to advance, regulatory frameworks must continue to evolve in parallel to fully support innovation in women’s health.

Ensuring that development frameworks support — rather than inadvertently slow — innovation is critical as this space continues to mature.

Looking ahead, where is the greatest opportunity over the next 5–10 years?

Kathleen:

The opportunity is in harnessing the innovation that’s already happening.

At women’s health conferences and scientific forums, we encounter and hear from incredibly bright founders and researchers tackling complex problems — premature ovarian insufficiency, endometriosis, polycystic ovarian syndrome, pregnancy-related complications. The ideas are strong. The science is advancing. The energy is measurable, and the momentum is accelerating .

What’s needed now is alignment — pairing that innovation with thoughtful development strategy, operational expertise, and sustained investment. The brainpower is in the room. The opportunity is to connect it to execution and scale.

Stacey:

I would add that the next phase of progress will depend on integrating patient voice and regulatory strategy earlier and more intentionally.

The growing strength of women’s voices is reshaping expectations for how products addressing women’s unmet health needs should be developed. We’ve seen programs succeed from a regulatory standpoint but fall short commercially because they didn’t fully reflect what women themselves identified as meaningful unmet need. Over the next decade, the opportunity is to design development programs that are scientifically rigorous, regulatorily sound, and genuinely responsive to women’s lived experiences. When patient voice and regulatory strategy guide development from the onset, therapies can do more than satisfy requirements; they can create real, lasting impact for the women who depend on them.

If we can align innovation, regulatory evolution, and real-world patient priorities, the impact in women’s health could be transformative.

To hear more about Kathleen’s and Stacey’s perspectives or to discuss how they can help shape your next women’s health program, contact them here.

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Premier applies a specialized approach to women’s health research that recognizes the unique impact of disease and treatment on women. With understanding built over more than three decades of dedicated women’s health experience, we deliver female centric trials that address the specialized clinical and regulatory considerations of women. 

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REFERENCES:

[i] The WHAM Report: The Business Case for Accelerating Women’s Health Investment (January 2026); RAND Corporation; McKinsey Health Institute.

[ii] McKinsey & Company. Closing the Women’s Health Gap: A $1 Trillion Opportunity to Improve Lives and Economies. 2024.