P108: A Targeted Literature Review Exploring Solutions for Inclusivity of Transgender and Non-Binary Patients in Clinical Research
Associate Director, Senior Consultant of Management Development
Parexel United States
The aim of this study was to review the current clinical development landscape to explore solutions for greater inclusivity of transgender and non-binary patients in clinical research.
A literature search was conducted between July 2018 to February 2022. Articles were included if they reported results from a clinical study that included transgender participants or results from analyses exploring the barriers and/or challenges to transgender clinical study participation.
During the time period reviewed, only 78 clinical studies were identified that included transgender participants. Most of these studies focused on either sexually transmitted HIV treatment or the effects of gender-affirming treatments. No other interventional randomised clinical studies reported the involvement of transgender patients.
A broad literature review was conducted to further explore the barriers contributing to the limited transgender and non-binary patient representation in clinical studies. The search identified barriers directly involving the patient community, the clinical sites overseeing the trials and regulatory bodies. From a patient perspective, reported barriers included a lack of trust in the medical profession and the pharma industry, intentional and non-intentional discrimination, site infrastructure concerns such as a lack of gender-neutral restrooms, and additional burden of trial participation demands. From a clinical site perspective, the dearth of formalized healthcare training and guidance emerged as key barriers; for example, the absence of hormone dosing guidance or hormone therapy effect on overall physiology in medical practice and associated safety concerns, no reference ranges for laboratory tests, and the challenge of defining gender operationally for research purposes. The challenge of recruiting enough patients for rigorous statistical analysis was reported to contribute to these barriers. Of note, a review of regulatory guidance globally failed to identify specific information related to transgender and non-binary patient participation in clinical research. The FDA’s 2020 report on enhancing the diversity of clinical trial populations stated that the agency recognized the distinction between gender and sex but that it was outside of the scope of the guidance to comment further, and EMA regulation 536/2014 on clinical trials on medicinal products for human use made no distinction between gender and sex.
To date, there is a lack of guidance on safe and inclusive practices for transgender and nonbinary individuals in clinical research. Whilst some areas such as practical challenges can be easily addressed, a lack of information around dosing best practices highlights a potential safety risk, which must also be balanced against a broader ethical consideration around fair and equitable access to potential new medications. More research is therefore needed to bridge this gap, alongside country-specific regulatory guidance. Together, these actions could help foster a more inclusive research approach that is more representative of the wider population.