The recent FDA approvals of isatuximab (Sarclisa) and daratumumab (Darzalex) combination therapies for first-line multiple myeloma treatment have spotlighted a persistent crisis in clinical research: the severe underrepresentation of minority patients in pivotal trials.
The phase 3 IMROZ and PERSEUS trials, which evaluated these treatments in combination with bortezomib, lenalidomide, and dexamethasone, included Black patient participation rates of just 0.9% and 1.3% respectively. These figures stand in stark contrast to the fact that Black individuals comprise 20% of newly diagnosed multiple myeloma patients and 14% of the US population.
Broader Pattern of Underrepresentation
A comprehensive 2020 FDA Center for Drug Evaluation and Research study examining 32,000 clinical trial participants revealed systematic disparities in trial participation:
- Non-Hispanic Whites: 75% of trial participants (vs. 59% of US population)
- Hispanic participants: 11% (vs. 19% of US population)
- Black/African American participants: 8% (vs. 14% of US population)
Impact on Treatment Outcomes
The lack of diverse representation has significant implications for real-world treatment effectiveness. Clinical trial populations tend to be younger, healthier, and less diverse than the general patient population, leading to a concerning disparity in outcomes. Studies have shown that real-world multiple myeloma patients experience 44% worse progression-free survival compared to clinical trial participants.
Barriers to Diverse Participation
Several factors contribute to low minority participation in clinical trials:
- Inaccessible trial locations
- Strict exclusion criteria
- Lower participation from patients of lower socioeconomic status
- Underrepresentation of older patients and those with comorbidities
Proposed Solutions
Healthcare industry experts recommend several approaches to address these disparities:
- Implementation of decentralized trials with multiple locations to improve accessibility
- Utilization of artificial intelligence for better patient-trial matching
- Broadening eligibility requirements with fewer exclusions
- Creating incentives for manufacturers to increase trial diversity, including expedited reviews and fee waivers
- Engaging community champions and navigators to educate patients about clinical trials
Industry Response
Organizations like Evolent are implementing new evaluation protocols, including health equity assessments for clinical trials that examine population representation and outcome equality across patient subgroups. They are also incentivizing healthcare providers to increase patient enrollment in clinical trials.
The path forward requires a concerted effort to ensure clinical trials truly represent the diverse patient populations who will ultimately receive these therapies. Only through more inclusive research can we ensure optimal treatment outcomes for all patient groups.
