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

In recognition of Women’s History Month, we spoke with a leader who brings complementary perspectives to women’s health development: Stacey Ayres, PhD, Vice President, Regulatory Strategy.

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

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.

Where have you seen the most meaningful progress in women’s health research over the past decade?

Stacey:

We’re 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?

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.

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?

Stacey:

I would say 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.

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

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.

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

ABOUT PREMIER RESEARCH:

Premier Research International LLC (Premier) is a leading global clinical research organization (CRO) and consulting partner with expertise in driving an efficient and effective path to market for the life sciences industry. Built with the needs of biotech in mind, our integrated approach helps life science innovators turn breakthrough science into life-changing drugs, devices, and diagnostics by addressing trial complexity, overcoming development hurdles, and demonstrating product value. 

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. 

Explore our women’s health clinical research expertise.

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.