July 13, 2023, 10:53 AM EDT
Attempts at improving diversity, equity, and inclusion (DEI) access across U.S. healthcare is a much-welcomed, if not long-overdue, development in recent years. Substantive DEI legislation was signed into U.S. law in December 2022 (and went into effect in February 2023), helping to correct significant disparities (explained below) and holding sponsors responsible for ensuring that their trials include participants from diverse groups. As we learned from vaccine development during the pandemic, clinical trials must address diverse participant populations to determine the real-world impacts on safety and efficacy for novel therapies. Data analytics tools can help.
Clinical trial participation among minority groups in the U.S. remains woefully inadequate. For example, a 2022 report by the National Academies of Sciences documents that Hispanic and African-American participants have historically accounted for less than 10% of all participants in cancer clinical trials, even though these populations make up a far greater percentage of cancer patients in the overall U.S. population. According to the FDA, Black, Hispanic, and Asian participation lagged far behind Caucasian participation in trials for drugs approved between 2015 and 2019. Similarly, women, LGBTQ individuals, older adults, individuals with disabilities, and many others who are often disproportionately impacted by specific health conditions are not well represented in clinical trials addressing their conditions.
While this information is not surprising, it should nonetheless be cause for alarm. Beyond the obvious notions of ensuring fairness and respect for all individuals regardless of their race, gender, sexual orientation, age, religion, ability, or any other personal characteristic, these efforts will drive many additional benefits such as reducing outcome bias, which may increase the risk of adverse events and the approval of therapies that are ineffective in marginalized populations. For example, one top reason for FDA drug recalls is adverse events in women.
No one doubts the inherent value of broader representation in clinical trials. However, pharmaceutical sponsors are uncertain of what these new regulations mean for upcoming clinical trials. One concern, according to various conversations with research professionals, is that such regulation might impact their ability to carry out any research at all.
“Everyone realizes the benefits of representative populations as participants in clinical trials,” said one multinational pharmaceutical company clinical operations executive in an April 2023 interview. “Now the issue has more to do with how granular regulations might get, how it fits into overall drug development strategies, and whether it might ultimately hinder our ability to address the requirements across all regions of development while controlling for costs and ensuring scientific rigor.”
Sponsors will need a reliable mechanism to ensure the representation of diverse participants in their trials, or risk potentially negative consequences. For example, one organization faced slowdowns during its COVID-19 vaccine trials due to a failure to recruit enough minority participants. With this new regulation in place, the risks are bigger and impact even more consequential.