History lesson

The science of drug development is only as strong as the evidence behind it. In women’s health, that evidence has long been limited in scope.

For decades, women were significantly underrepresented in clinical trials. Recent years have seen improvement, but there are lingering gaps in our collective understanding of how therapies work across sexes, life stages, health conditions and care settings.

This is partly because the design and execution of clinical trials have sometimes overlooked the unique factors that influence women’s health, such as hormonal fluctuations, caregiving roles and health conditions specific to women.

This underrepresentation has consequences. Conditions like menopause, endometriosis and autoimmune disorders, to name a few, remain under-researched. The result is an evidence base that doesn’t always reflect the needs of half the population.

Maternal health illustrates this gap in especially urgent terms.

Approximately 200 million women become pregnant globally each year, yet only a few medications have been specifically developed and approved for use during pregnancy in the last 30 years.

In fact, roughly 90% of pregnant women report taking some type of medication, yet fewer than 10% of medicines approved since 1980 have sufficient information to determine their safety during pregnancy. This lack of dedicated research leaves clinicians without clear, evidence-based guidance – and patients without confidence in their treatment options.

Many of the historical barriers to including women are well known: long-standing concerns about safety and liability, often tracing back to high-profile incidents like the thalidomide crisis of the 1950s.

In response, the pendulum swung towards extreme caution. While that caution was understandable, it created an enduring pattern of exclusion that still exists today.

And in the absence of robust, representative data, pregnant and breastfeeding individuals are left to navigate complex health decisions without the clarity that rigorous science should provide.

Ask any mother about her experience using medication while pregnant, and you’ll likely hear the same refrain: check the label; consult your doctor and hope for the best. But hope is not a substitute for evidence.

Improving trial representation in clinical research, particularly for women, requires multi-stakeholder collaboration. While there is no one-size-fits-all solution, several strategies are helping move the needle in the right direction:

1. Design trials that reduce the participation burden
Many women face logistical or personal barriers that can make trial participation difficult. Decentralised and pragmatic trial designs can reduce this burden by allowing more flexible participation – something that can be especially important during pregnancy or early parenthood.

These approaches not only increase enrolment but also expand the geographic and demographic diversity of participants.

2. Gather patient input early and often
Tools that enable early engagement with patients – such as TransCelerate’s Patient Protocol Engagement Toolkit (P-PET) and Study Participant Feedback Questionnaire (SPFQ) – help trial sponsors incorporate patient perspective throughout the study process.

These insights can lead to more comprehensive, accessible studies by identifying and addressing potential hurdles before the first participant is enrolled. Making space for lived experience in protocol development leads to stronger study design and more reliable results.

3. Embed inclusion in trial design frameworks
Findings from a regulatory landscape assessment focused on pregnancy and breastfeeding were recently used to inform updates to a protocol template framework designed to support consistent and inclusive trial documentation.
The resulting Clinical Template Suite includes an integrated participant type library for trials enrolling pregnant participants to address regulatory gaps and support better trial design.

4. Shift from exclusion to thoughtful inclusion
In the past, excluding pregnant and breastfeeding individuals from clinical trials by default was often seen as the safest path forward. Today, there is growing recognition that clinical trials that include pregnant and breastfeeding women – guided by appropriate protocols and ethical oversight – can lead to effective evidence and safer care.

This shift in mindset, from risk avoidance to thoughtful inclusion, can help ensure that maternal health needs are considered earlier in trial planning, rather than as an exception after the fact.

The practical application of these tools and insights is beginning to shape how trials are designed and run.

In one example, a pharmaceutical company used findings from TransCelerate’s pregnancy and breastfeeding landscape assessment to revise its global clinical and post-marketing pregnancy follow-up form.

The result was a 40% reduction in data fields, streamlining the experience for participants while maintaining alignment with global regulatory requirements.

In another case, a sponsor used the same assessment to evaluate its pharmacovigilance processes, ensuring they aligned with the most current expectations across health authorities.

These incremental changes matter – they help reduce friction, increase confidence and contribute to more well-rounded research that will lead to more effective healthcare.

Progress in women’s health research cannot happen in a vacuum. Many of the most meaningful changes are the result of shared insight and collective action. Industry organisations, regulatory bodies, advocacy groups and academic institutions all play a role in shaping a more diverse research environment.

That collaboration can take many forms – from sharing tools and frameworks to contributing insights that inform future guidance to working together on cross-sector initiatives that expand access and representation.

In many instances, these partnerships have helped surface long-standing gaps, validate the need for clearer policies and lay the foundation for broader change.

Two examples of this type of ecosystem alignment include the ongoing work around ICH E21, which is a working group developing global guidance for the inclusion of pregnant and breastfeeding individuals in clinical research, and the ConcePTION project in Europe, a multi-stakeholder initiative focused on improving evidence generation and communication around the use of medicines in pregnancy and lactation.

While not always visible to the public, this kind of behind-the-scenes coordination drives true progress. It ensures that solutions are not developed in silos, but instead reflect the input and priorities of different stakeholders.

When the research community moves together, the impact reaches far, paving the way for more equitable and effective care.

There’s no question that researching how medications impact women, particularly those who are pregnant or breastfeeding, presents complex challenges. But that doesn’t mean it can’t be done.

For sponsors, it means exploring ways to design studies that are not only scientifically sound but also patient-informed. For regulators, it may involve continued efforts to provide clearer guidance and reduce fragmentation across geographies. For healthcare providers, it’s about advocating for stronger evidence to support shared decision-making with patients.

Perhaps most importantly, it’s about moving toward a model of research that sees women’s participation in clinical research and clinical trials not as an exception, but as an expectation.

Jeneen Donadeo is Executive Director, Portfolio Management at TransCelerate BioPharma