Disparities in African-American patient enrollment in pivotal phase 3 immunotherapy trials that led to FDA drug approval in patients with non-small cell lung cancer.

Abstract

99 Background: Blacks/African American have historically been underrepresented in enrollment into clinical trials. This leads to uncertainty regarding the efficacy and tolerability of treatments in this patient population following drug approval by the FDA. One of the approval criterion for the FDA is the patient population represents the diversity of patient population in the US as argued by the US FDA in the ORIENT-11 trial (NCT03607539). We abstracted the percentage of African-American into clinical enrolled into pivotal randomized phase 3 trials that led to approval of immunotherapy in advanced NSCLC. Methods: We abstracted the racial composition of randomized phase 3 trials that led to immunotherapy approval including neoadjuvant, adjuvant, consolidation/maintenance, first line and second line indication of immunotherapy in the U.S. Results: We identified a total of 20 randomized phase 3 trials that led to immunotherapy indication in NSCLC by the FDA including neoadjuvant (N = 1), adjuvant (N = 2), consolidation/maintenance (N = 1), first-line (N = 12), and second line (N = 4). We excluded 3 trials (ENPOWER-Lung01, -Lung03, and Checkmate-227) that did not report the racial composition of the patient participants. A total of 12490 subjects participated in the 17 trials analyzed. Of these, 66.3% were male and 33.7% were female. Distribution by race revealed 75.7% White (range 55.9% - 94.3%); 1.9% African-American (range 0.03% - 3.6%); 18.6% Asian (range 1.3% - 34.6%); and 3.8% (0.5% - 7.6%) were either mixed race, American Indian, Pacific Islander or unknown. Conclusions: African-Americans comprised an estimated 13.6% of the U.S. population respectively but our survey indicated only 1.9% of the trial population that led to immunotherapy approval in the US were African-Americans. The patient population in the clinical trials leading to immunotherapy regimen approval in the U.S. significantly under-represented African-Americans and here the true efficacy of and potential unknown adverse events of immunotherapy in African-Americans may over- or under-estimated. The U.S. FDA has the power to approve immunotherapy trials that are more representative of the US population.