Pregnant Women & Clinical Trials: Addressing Research Gaps & WHO’s 2030 Goal

For decades, pregnant and breastfeeding women have been largely excluded from medical research, creating significant gaps in our understanding of how medications and vaccines affect both mother and child. This historical underrepresentation means many expectant and new mothers are left navigating healthcare without evidence-based guidance, often relying on treatments used “off-label” – meaning for conditions they weren’t specifically approved for. A recent analysis, spearheaded by the Global Observatory on Health Research and Development, reveals that only 4% of clinical trials conducted over the past decade included pregnant women. This disparity isn’t simply a matter of fairness. it’s a critical public health issue with far-reaching consequences.

The World Health Organization (WHO) is now actively working to address this imbalance. Recognizing the urgent need for change, the WHO has established a global task force with the ambitious goal of achieving the timely and ethical inclusion of pregnant and breastfeeding women in clinical research by 2030. But achieving this goal requires a concerted effort from all stakeholders involved in the clinical trial process, from regulatory agencies to pharmaceutical sponsors and healthcare providers.

Why Have Women Been Historically Underrepresented?

The exclusion of pregnant women from clinical trials isn’t rooted in malice, but rather in a history shaped by a single, devastating event: the thalidomide tragedy of the late 1960s. The drug, prescribed for morning sickness, caused severe limb malformations in babies born to mothers who had taken it. This catastrophe understandably led to heightened caution and a widespread reluctance to expose pregnant women to experimental therapies. However, this caution has arguably become overly restrictive, creating a legacy of data gaps that continue to impact maternal and fetal health today.

Sheila Diamond, a digital health strategist at Medidata, emphasizes that the issue extends beyond simply protecting vulnerable populations. “It wasn’t until 1993 in the US that women were even allowed to be included or participate in clinical trials,” she notes. “We continue to navigate this systemic bias in health care.” Diamond explains that historically, medicine operated under the assumption that male physiology could represent humanity as a whole, leading to clinical trials that often restricted eligibility criteria for women and failed to adequately analyze sex-specific differences. This assumption overlooks crucial biological differences – chromosomal variations, hormonal fluctuations and variations in drug metabolism – that can significantly impact how women respond to treatments.

Beyond Pregnancy: Addressing Broader Gender Disparities

The underrepresentation of women in clinical trials isn’t limited to pregnant individuals. Women are often underrepresented in research across a wide range of health conditions, including cardiovascular disease – the leading cause of death for women globally – and Alzheimer’s disease, which affects nearly two-thirds of the seven million Americans living with the condition. Autoimmune diseases, which disproportionately affect women, too suffer from a lack of dedicated research. This lack of representation can lead to delayed diagnoses, inappropriate treatments, and poorer health outcomes.

The Complexities of Including Pregnant Women in Trials

Including pregnant women in clinical trials presents unique challenges. Beyond the ethical considerations, there are logistical hurdles related to monitoring both maternal and fetal health, as well as concerns about potential risks to the developing fetus. However, experts argue that excluding pregnant women isn’t necessarily the safest approach. “Is it more protective to exclude pregnant women, or is it to design more thoughtful stage inclusion strategies that generate some real evidence?” Diamond asks. The lack of data means that healthcare providers often have to produce treatment decisions based on limited information, potentially exposing pregnant women to unnecessary risks.

Fortunately, there’s growing momentum towards more inclusive trial designs. Researchers are exploring adaptive trial designs, which allow for adjustments to study protocols based on real-time data, and leveraging pregnancy exposure registries to collect information on the safety of medications during pregnancy. The responsible use of artificial intelligence (AI) and advanced analytics is also playing a role, helping researchers to identify safer inclusion criteria and evaluate potential risks more effectively. Real-world evidence is becoming increasingly important in this context.

A Collaborative Approach is Essential

The WHO’s call to action underscores the need for a collaborative approach to address this issue. It’s not solely the responsibility of the WHO or regulatory agencies; sponsors, physicians, scientists, and even trial patients all have a role to play. Sponsors can implement smarter trial designs that prioritize the inclusion of pregnant women, while regulators can provide clearer ethical and regulatory guidance. Increased investment in women’s health research is also crucial.

Diamond emphasizes the importance of multi-stakeholder collaboration. “It’s not just one side of the house; it’s everyone collectively working together, ensuring that their diverse thoughts and input are integrated into this wider remit.”

What’s on the Horizon for More Inclusive Trials?

Several promising strategies are gaining traction. These include incorporating more adaptive trial designs, utilizing pregnancy exposure registries to gather real-world data, and leveraging advanced analytics and simulation modeling to assess potential risks. The WHO is also encouraging the development of clearer ethical and regulatory guidelines to facilitate the inclusion of pregnant and breastfeeding women in clinical research. The goal is to move beyond a culture of exclusion and embrace a more data-driven, inclusive approach that prioritizes the health and well-being of all mothers and their babies.

You can find more information about the Global Observatory on Health Research and Development on the WHO website. If you are pregnant or breastfeeding and have questions about medications or vaccines, please consult with your healthcare provider.