A new focus on women’s health: Exploring the EMA’s latest initiatives

The European Medicines Agency (EMA) has recently launched a “Women’s health” landing page on its website (here) to bring together information on a range of topics and that should help to advance women's health in the context of the development, evaluation and use of medicines. The initiatives reflect a growing recognition that medicines regulation has an important role to play in addressing long-standing gender disparities in healthcare. They also support innovation in the women’s health space. Some of the key areas covered on the landing page are summarised below.

At the centre of the EMA’s approach is a move away from the historic ‘male by default’ model of medicines development. Accordingly, a key area of focus is improving the participation of underrepresented populations in clinical trials, including women, and pregnant and breastfeeding individuals. The EU Clinical Trials Regulation requires that trial enrolment reflects the populations likely to use the medicine under investigation. This means that where a disease disproportionately affects women, the trial population must reflect this reality. The EMA has also indicated that its clinical trial infrastructure, including the Clinical Trials Information System (CTIS), will enable more detailed analysis of representation across different stages of clinical development and across population groups, helping to identify and address gaps in female participation.

With respect to the development of medicines aimed at addressing women’s health needs, the EMA is encouraging companies to engage with them early using the existing early development support tools. Available resources include scientific advice procedures, academia briefing meetings, and dedicated assistance for small and medium-sized enterprises.By promoting early regulatory engagement, the EMA aims to help sponsors address scientific and methodological challenges relating to women’s health before they become obstacles to development.

Pregnancy and breastfeeding are another key focus of the EMA’s initiatives. Historically, pregnant and breastfeeding women have often been excluded from clinical research, resulting in limited evidence regarding the safety and efficacy of many medicines in these populations. Healthcare professionals and patients are often then left to make treatment decisions in the face of incomplete evidence. In response, the EMA is promoting various initiatives to help produce information throughout the lifecycle of medicines on the benefits and risks of the use of medicines during pregnancy and breastfeeding. For example, the EMA has developed an algorithm within EudraVigilance to detect adverse drug reactions during pregnancy. The EMA has also produced pregnancy and breastfeeding specific guidance. For example, it worked with international partners on programmes such as the ICH E21 guideline, which aims to support the inclusion of pregnant and breastfeeding women in clinical trials where appropriate.

Another aspect emphasised by the EMA is the need to generate real-world evidence relating to women’s health. Through DARWIN EU® (Data Analysis and Real World Interrogation Network), the EMA commissions studies on disease patterns, disease burden and medicine use in women. The areas of research include the use, safety and efficacy of medicines in women, as well as female-specific conditions such as endometriosis. These studies aim to contribute towards closing the women’s health data gap, particularly where limited information is available from clinical trials.

Taken together, the EMA’s women's health initiatives reflect a broader shift in thinking about how medicines should be developed, evaluated and monitored. Rather than treating gender differences as a secondary consideration, the EMA is seeking to ensure that women's health is explicitly taken into account at every stage of a medicine’s lifecycle, from clinical development and regulatory review to post-authorisation evidence generation and safety monitoring.