BoMedAC®Sex-based representation in clinical research
Why the inclusion of women and sex-specific analyses matter for drug development, and what regulators such as the FDA expect.
Men and women differ in ways that can affect how diseases progress and how drugs work. Yet clinical trials historically excluded women, leaving gaps in what we know about efficacy and safety for half the population. Regulators – including the US Food and Drug Administration (FDA) – now expect sponsors to address this.
Why it matters
- Biology: differences in pharmacokinetics, hormones and disease pathophysiology can change efficacy and safety.
- Generalisability: results from trials that under-represent women may not apply to real-world patients.
- Equity: insufficient representation contributes to health disparities.
What regulators expect
The FDA encourages the inclusion of women in clinical trials, provides guidance on studying and analysing sex differences, reviews sex-specific data in new drug applications, and expects labelling to include sex-specific information where it is clinically relevant. Real-world evidence and post-marketing surveillance complement trial data on sex differences.
Practical strategies
- Recruitment: targeted strategies; accessible sites and scheduling, including childcare and transport.
- Transparency: communicate purpose, risks and benefits clearly to build trust.
- Data: collect sex-specific demographic, clinical and biomarker data, including hormonal status where relevant.
- Analysis: plan sex-specific subgroup analyses with appropriate statistical methods, and report the findings transparently.
Further reading
Vijayasingham L, Bischof E, Ateghang-Awankem B, et al. Acting on sex and gender in medical innovation is good for business. BMJ 2023;381.
Dr. Bernadette Awankem
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