AML-406: Age-Based Disparities in Clinical Trials Supporting Food and Drug Administration (FDA) Approval of Therapies for Hematologic Malignancies

Abstract

Context: The largest proportion of patients diagnosed with hematologic malignancies is older adults, defined as age ≥65. The under-representation of older adults in oncology clinical trials has been previously described; however, data specific to hematologic malignancy trials is sparse. Objective: To evaluate the median age of clinical trial participants and compare it to the real-world age distribution for each respective malignancy. Design: Prescribing information for novel hematologic malignancy therapies first approved for an adult indication between 2015 and 2019 were reviewed for the median age in the evaluable population of the supporting clinical trials. Adult median age estimates for each indication were obtained from the SEER database. Difference in median age (DMA) was calculated by subtracting disease population median age from trial median age. Additional trial protocol details were obtained from FDA package labels. Data are presented using descriptive statistics. Results: Twenty-two hematologic malignancy therapies were approved based on 26 trials, with 6,033 patients in the evaluable populations. Less than half of the trials were phase III (10; 38%) or were randomized with a control group (11; 42%). The median age of the trial participants was younger (mean 3.8 years) than the median age of the disease populations (95% CI: -6.0 to -1.6 years; p Conclusion: The median age of trial participants was nearly 4 years younger than their respective disease populations, despite no age-based exclusions in most protocols. Poor performance status, comorbidities, or organ impairment likely contributed to older patients’ ineligibility. Adequate representation in clinical trials is critical to evaluate the safety and efficacy of new therapies in the patient population who will eventually receive them. Post-approval, real-world research can help understand the effect of these therapies on older adults.