EMA moves to close long-standing gaps in women’s medicines development
The European Medicines Agency is preparing a regulatory roadmap aimed at changing how women’s health is considered across medicines development and oversight, with publication planned by the end of 2026. The effort follows a two-day multistakeholder workshop in Amsterdam on 28 and 29 September, where regulators, patients, clinicians, industry, academia, and NGOs aligned on a set of priority actions.
The clinical stakes are obvious. When women are underrepresented in evidence generation, the result is not a theoretical imbalance; it can shape dosing decisions, safety monitoring, and the interpretation of benefit-risk profiles for years after a product reaches the market.
EMA framed the initiative as a response to persistent gaps in research and development that leave many women’s health needs insufficiently understood. The agency’s executive director, Emer Cooke, said women are still missing from research and data, prevention, diagnosis, and treatment, and pointed to the workshop as evidence of what can happen when diverse voices converge on the same problem. That message was paired with a practical promise: the agency will use the dialogue to build a roadmap that can drive progress in Europe and beyond.
At the center of the discussion was a basic regulatory question with far-reaching consequences: whether current medicines development reliably captures sex- and gender-related differences in disease presentation, treatment response, and adverse effects. In too many programs, women remain inadequately enrolled, and the resulting datasets may fail to reflect pregnancy, breastfeeding, age-related physiologic changes, or life-stage-specific needs. The consequence is a thinner evidence base at precisely the point where clinicians need clarity most.
Ten priority actions, not one symbolic gesture
Over the course of the workshop, participants prioritized 10 proposed actions that fall into areas where EMA could lead, collaborate, or influence. The list is expansive, but the logic is consistent: better medicine for women requires earlier identification of evidence gaps, stronger data collection, and more disciplined reporting of sex-disaggregated results.
The priorities begin with collaboration. EMA and stakeholders are expected to work together to define unmet needs and research gaps, then support research with adequate funding to address them. From there, the roadmap pushes toward better trial design and better enrollment: increasing women’s participation in clinical trials, improving reporting of high-quality sex-disaggregated data, and supporting the inclusion of pregnant and breastfeeding individuals in studies where appropriate.
The inclusion of pregnancy and breastfeeding in the conversation is particularly consequential. Excluding these groups by default may simplify trial logistics, but it also leaves clinicians with sparse evidence when real-world prescribing demands decisions during periods of rapid physiologic change, altered pharmacokinetics, and heightened concern about fetal or neonatal exposure.
Women’s health across the life course also figured prominently. Participants called for evidence generation that reflects women of all ages and stages of life, not just the narrow populations that often dominate registration studies. That approach is clinically important because disease burden, hormonal milieu, and medication tolerability can differ substantially across adolescence, reproductive years, perimenopause, and older age.
Data quality, real-world evidence, and regulatory literacy
The roadmap is not limited to trial enrollment. It also calls for a sustainable infrastructure for collecting and analyzing real-world data, a training effort for the European medicines regulatory network on sex- and gender-sensitive assessment, and stronger communication of benefits, risks, and uncertainties related to medicines use in women.
Real-world data can fill some of the evidence gaps left by clinical trials, especially when trial eligibility criteria exclude common comorbidities or life-stage situations. Yet those datasets only help if they are structured well enough to support careful analysis and if the questions being asked are biologically meaningful. Sex- and gender-sensitive assessment training may sound procedural, but the downstream effect could be substantial: regulators who are better equipped to recognize differences in symptom patterns, pharmacology, and adverse-event signals are more likely to ask the right questions before approval and after market entry.
María Lamas, director of the Spanish Agency for Medicines and Health Products and chair of the Heads of Medicines Agencies’ Management Group, argued that meaningful change starts with dialogue and a shared understanding of the challenge. She said one key priority is proper representation of women in clinical trials and data that capture their experiences and needs, so that evidence can support informed decisions and better care. The emphasis on informed decision-making is not rhetorical. In regulatory medicine, poor representation can distort the estimate of both benefit and harm, and clinicians are then left extrapolating from populations that do not fully match the patients in front of them.
The workshop drew more than 100 voices from Europe and internationally, spanning regulators, patients, healthcare professionals, NGOs, industry, and academia. That range matters because no single stakeholder group can repair the evidence deficit alone. Trial sponsors control protocol design, investigators influence recruitment, regulators shape evidentiary standards, and patients determine whether studies are trustworthy enough to join.
What the roadmap will have to deliver
EMA said the priority actions will feed into a roadmap on medicines for women’s health that will include concrete deliverables with short-, medium-, and long-term timelines. The roadmap and the workshop report are planned for publication by the end of 2026.
A framework of measurable outcomes and key performance indicators sits near the end of the list, but it may prove central to the entire exercise. Without metrics, commitments to equity can become ceremonial. With them, regulators can track whether women are entering studies at higher rates, whether sex-disaggregated reporting is improving, whether pregnancy and breastfeeding evidence is expanding, and whether communication about uncertainty is becoming clearer in labeling and guidance.
The agenda is ambitious because the problem is structural. Women have historically been left behind not only in trials, but also in the broader machinery that determines which questions are asked, which outcomes are prioritized, and which data are considered sufficient for decision-making. A roadmap will not erase those patterns on its own, yet it can redefine the expectations that sponsors and regulators must meet if medicines for women are to rest on evidence rather than assumption.
The next inflection point is concrete rather than symbolic: the planned publication of the roadmap and workshop report by the end of 2026, followed by whether the proposed KPIs and timelines can translate an overdue policy consensus into routine regulatory practice.