Women’s Health and Clinical Evidence: What EMA’s Growing Regulatory Focus Means for Medicines Development

On September 28–29, 2026, the European Medicines Agency (EMA), together with partners in the European Medicines Regulatory Network, convened a multi-stakeholder workshop dedicated to women’s health. The meeting brought together regulators, patients, healthcare professionals, academia, industry, and global partners to examine existing evidence gaps, identify research priorities, and consider opportunities to strengthen medicines development and clinical research.

The discussion is important for pharmaceutical and biotechnology organizations because women’s health is not limited to medicines developed specifically for conditions affecting women. It also involves how diseases present differently across populations, whether clinical trial populations appropriately reflect the people expected to use a medicine, how sex-related differences are evaluated, and how evidence is generated throughout the medicine lifecycle.

For Regulatory, Clinical, Safety, Medical, and development teams, these considerations increasingly reinforce the importance of building representative evidence into development strategy from the beginning.

Women’s Health Is Broader Than Women-Specific Indications

Discussions about women’s health in drug development are sometimes associated primarily with reproductive health, pregnancy, or conditions that predominantly affect women.

EMA’s current work takes a broader perspective.

The Agency has identified continuing gaps in understanding how diseases and medicines may affect women and men differently, while also recognizing the need for improved treatments for conditions that primarily affect women. These considerations can influence clinical development, benefit-risk assessment, safety monitoring, labeling, and post-authorization evidence generation.

For sponsors, this means that questions related to sex, gender, age, life stage, pregnancy, breastfeeding, and other relevant population characteristics may need to be considered as part of the overall evidence strategy rather than treated as isolated issues late in development.

Representation in Clinical Trials Is Already a Regulatory Consideration

An important distinction for sponsors is that representative clinical trial populations are not simply an emerging policy concept in Europe. Under the EU Clinical Trials Regulation, clinical trial participants are expected to represent the populations likely to use the medicine. EMA specifically notes that when a disease is more common in women, the trial population needs to reflect that population.

EMA also monitors participation in clinical trials across population groups, including women, and information on the representation of women and men in trials authorized under the Clinical Trials Regulation is included in monitoring of the European clinical trials environment.

The Clinical Trials Information System is also expected to support more detailed analyses of representation across clinical development, population groups, life stages, age groups, and ethnicities. For development organizations, this makes trial population strategy an important planning consideration. Enrollment should not be viewed only as an operational challenge. The population selected for a clinical program ultimately affects how confidently the resulting evidence can support conclusions about the population expected to receive the medicine.

Sex-Disaggregated Data Can Strengthen the Evidence Base

Enrollment alone does not answer every question about representativeness.

Understanding whether clinically meaningful differences exist may also require appropriate analysis of data by sex and other relevant characteristics.

EMA states that EU regulators assess differences between women and men in the efficacy, safety, and use of medicines before authorization where relevant. When those differences have implications for how a medicine should be used, they may be reflected in product information. After authorization, evidence generation continues. Companies are expected to monitor the safety and efficacy of their medicines, and EMA notes that data broken down by sex should be considered where relevant and available, including important findings arising from company-sponsored clinical trials, scientific literature, and spontaneous adverse-reaction reporting.

For sponsors, the practical consideration is not simply whether data can be separated by sex after a study is complete. The more important question is whether the development program was designed to generate data capable of answering the relevant clinical and regulatory questions. That consideration may affect enrollment strategy, statistical planning, data collection, safety evaluation, and ultimately the interpretation of benefit and risk.

Pregnancy and Breastfeeding Require Lifecycle Evidence Planning

Pregnant and breastfeeding individuals present a particularly important evidence challenge.

Historically, these populations have frequently been excluded from clinical research, leaving healthcare professionals and patients with limited information when treatment decisions must be made in clinical practice.

EMA has been developing a broader strategy to improve evidence on the benefits and risks of medicines used during pregnancy and breastfeeding. This work extends across the medicine lifecycle, including preclinical research, clinical trials, post-authorization evidence generation, pharmacovigilance, and product communication. EMA is also contributing to the development of ICH E21, which addresses the inclusion of pregnant and breastfeeding individuals in clinical trials.

For sponsors, this does not mean that every clinical program should automatically enroll pregnant or breastfeeding participants. Decisions must remain scientifically and ethically appropriate and should reflect the medicine, indication, population, known and potential risks, and applicable regulatory requirements. It does, however, reinforce the need to consider early how evidence for these populations will ultimately be generated when the medicine may be used by them in clinical practice.

Real-World Evidence Has an Important Role

Clinical trials cannot answer every question that emerges across the lifecycle of a medicine.

Real-world data can therefore play an important role in understanding treatment patterns, effectiveness, safety, medicine utilization, and outcomes in populations that may be difficult to study adequately during pre-authorization development. EMA already uses real-world evidence infrastructure and studies to examine questions relevant to women’s health, including pregnancy and breastfeeding, medicine utilization, and sex-specific safety considerations.

This creates an opportunity for organizations to think about evidence generation as a continuum rather than separating clinical development from post-authorization activities.

A well-developed evidence strategy considers what must be established before authorization, what questions may remain after approval, and how post-authorization data can appropriately address those questions.

The Regulatory Strategy Should Start With the Intended Population

For Regulatory and Clinical teams, one of the most useful questions to ask early in development is straightforward:

Who will actually use this medicine? The answer should influence more than the demographic table in a clinical study report. It can affect eligibility criteria, recruitment strategy, study design, statistical considerations, safety monitoring, pharmacokinetic assessments, benefit-risk evaluation, labeling, and post-authorization evidence generation. When the intended treatment population includes meaningful differences across sex, age, reproductive status, or other characteristics, development teams should determine whether the evidence strategy is capable of addressing those differences. Waiting until submission preparation to identify an important evidence gap can leave organizations with limited options for resolving it.

Clinical Evidence Strategy Is a Cross-Functional Responsibility

Representative evidence is not solely the responsibility of Clinical Operations.

Regulatory Affairs may need to interpret regional expectations and determine how evidence supports the proposed indication and labeling.

Clinical Development defines the scientific questions and study strategy.

Biostatistics helps ensure the analyses can meaningfully address those questions.

Medical and Safety teams evaluate clinical relevance and benefit-risk considerations.

Clinical Operations must determine whether the planned population can realistically be recruited and retained.

Pharmacovigilance and real-world evidence teams may need to address questions that remain after authorization. Program leadership must connect these activities to development milestones, resources, dependencies, and decision points. When these functions work independently, evidence gaps may not become visible until late in the program. When they are integrated, population and evidence considerations can become part of development planning from the beginning.

What Pharmaceutical and Biotechnology Organizations Should Consider

EMA’s growing focus on women’s health provides an opportunity for organizations to review their current development practices. Teams should consider whether clinical trial populations appropriately reflect the patients expected to use the medicine and whether relevant differences by sex, age, or life stage have been considered in the evidence strategy.

Organizations should also evaluate whether pregnancy and breastfeeding considerations have been addressed where relevant, whether statistical and data strategies can support appropriate subgroup analyses, and whether post-authorization or real-world evidence may be necessary to address questions that cannot reasonably be resolved before approval.

Just as importantly, these considerations should be connected across Regulatory, Clinical, Medical, Safety, Biostatistics, Pharmacovigilance, and program management.

The objective should not be representation for representation’s sake. It should be the generation of scientifically meaningful evidence that supports an appropriate understanding of a medicine’s benefits and risks in the population for whom it is intended.

Looking Ahead

EMA’s September 2026 multi-stakeholder workshop is part of a broader effort to embed women’s health considerations more effectively into medicines development and regulation.

The workshop was designed to identify current gaps and challenges, explore opportunities for more inclusive and representative clinical research, and identify priority areas for future action. For industry, it will be important to distinguish between existing regulatory expectations and future initiatives that emerge from these discussions.

Not every priority discussed in a workshop represents a new regulatory requirement. However, these discussions can provide valuable insight into areas receiving increased regulatory attention and where evidence-generation practices may continue to evolve.

Regulatory intelligence is most useful when organizations do more than monitor these developments. The greater value comes from evaluating how relevant developments may affect existing development strategies, identifying potential gaps early, and determining whether program plans should evolve as the regulatory environment develops.

About OneGlobal Trinity M Consulting

OneGlobal Trinity M Consulting supports medical device, pharmaceutical, and biotechnology organizations navigating complex regulatory, quality, clinical, product development, validation, and program execution challenges. We work with organizations to connect strategy with execution, helping cross-functional teams align regulatory requirements, development activities, evidence needs, project timelines, risks, and key decisions throughout the product lifecycle.

Our experience across global life sciences programs enables us to support organizations as they navigate evolving regulatory expectations, complex development initiatives, and the operational challenges associated with bringing products to market.

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References

• European Medicines Agency. Women’s Health. EMA overview of initiatives addressing women’s health in medicines development and regulation.

• European Medicines Agency. Multi-stakeholder Workshop on Women’s Health. September 28–29, 2026.

• European Medicines Agency. Medicines During Pregnancy and Breastfeeding. EMA overview of regulatory and evidence-generation initiatives related to pregnancy and breastfeeding.

• European Union. Regulation (EU) No 536/2014 on Clinical Trials on Medicinal Products for Human Use.

• European Medicines Agency. Accelerating Clinical Trials in the EU (ACT EU). Initiatives addressing the European clinical-trial environment and inclusion of underrepresented populations.