Division of Hematologic Malignancies I, Center for Drug Evaluation and Research, U.S. Food and Drug Administration, Silver Spring, MD.
Division of Biometrics IX, Center for Drug Evaluation and Research, U.S. Food and Drug Administration, Silver Spring, MD.
Clin Lymphoma Myeloma Leuk. 2023 Jun;23(6):463-470.e1. doi: 10.1016/j.clml.2023.03.012. Epub 2023 Mar 30.
Patients of certain racial and ethnic groups have been underrepresented in clinical trials for treatment of malignancy. One potential barrier to participation is entry requirements that lead to patients in various racial and ethnic groups not meeting eligibility criteria for studies (ie, "screen failure"). The objective of this study was to analyze the rates and reasons for trial ineligibility by race and ethnicity in trials of acute myeloid leukemia (AML) submitted to the U.S. Food and Drug Administration (FDA) between 2016 and 2019.
Multicenter, global clinical trials submitted to the FDA to support AML drugs and biologics. We examined the rate of ineligibility among participants screened for studies of AML therapies submitted to the FDA from 2016 to 2019. Data were extracted from 13 trials used in approval evaluations, including race, screen status, and reason for ineligibility.
Overall, patients in historically underrepresented racial and ethnic groups were less likely to meet entry criteria for studies compared to White patients, with 26.7% of White patients, 29.4% of Black patients, and 35.9% of Asian patients not meeting entry criteria. Lack of relevant disease mutation was the reason for ineligibility more frequently among Black and Asian patients. The findings were limited by the small number of underrepresented patients screened for participation.
Our results suggest that entry requirements for studies may put underrepresented patients at a disadvantage, leading to less eligible patients and thus lower participation in clinical trials.
某些种族和族裔群体的患者在恶性肿瘤治疗的临床试验中代表性不足。参与临床试验的一个潜在障碍是入组要求，导致不同种族和族裔的患者不符合研究的入选标准（即“筛选失败”）。本研究的目的是分析在 2016 年至 2019 年期间向美国食品和药物管理局（FDA）提交的急性髓系白血病（AML）临床试验中，按种族和族裔分析试验排除的发生率和原因。
多中心、全球性临床试验，向 FDA 提交以支持 AML 药物和生物制剂。我们检查了 2016 年至 2019 年向 FDA 提交的 AML 治疗研究筛选参与者中排除的比例。数据来自用于批准评估的 13 项试验中提取，包括种族、筛选状态和排除原因。
总体而言，与白人患者相比，历史上代表性不足的种族和族裔群体的患者更不可能符合研究的纳入标准，26.7%的白人患者、29.4%的黑人患者和 35.9%的亚洲患者不符合纳入标准。缺乏相关疾病突变是黑人患者和亚洲患者排除的主要原因。由于筛选参与的代表性不足患者数量较少，因此研究结果存在一定的局限性。
我们的研究结果表明，研究的纳入要求可能使代表性不足的患者处于劣势，导致合格患者减少，从而降低了临床试验的参与度。
