Background: Evaluating clinical trial representation for countries with different socio-demographic index (SDI) and tyrosine kinase inhibitor (TKI) availability for chronic myeloid leukemia (CML).Methods: CML incidence rates (IRs) and disability-adjusted life years (DALYs) (1999–2019) from the Institute of Health Metrics and Evaluation were analyzed. Trials investigating TKI use in CML were obtained from ClinicalTrials.gov. Site data for eligible trials (N = 30) and DALYs were analyzed. TKI approvals, DALYs, and IRs were summarized by SDI.Results: North America (NA) had significant decreases in annual percent change (APC) in DALYs and incidence rates from 1999 to 2004. IRs were highest in Europe and Central Asia (ECA) and NA, while DALYs were highest in South Asia (SAsia) and Sub-Saharan Africa (SSA). Countries in the high-SDI quintile were likely to have lower DALYs than lower-SDI quintiles. Differences in regional DALYs vs. sites in TKI trials were significant for SAsia, SSA, and ECA. High-SDI countries were included in all 30 trials, and TKI approvals were prominent in high-SDI (142) vs. low-SDI (14) countries.Conclusions:The inclusion of disproportionately affected countries during the design of and recruitment into clinical trials should occur, as should TKI availability. The lack of representation demonstrates healthcare disparities.
背景：本研究旨在评估不同社会人口指数（SDI）国家在慢性髓系白血病（CML）临床试验中的代表性及其酪氨酸激酶抑制剂（TKI）的可及性。 方法：分析健康指标与评估研究所提供的1999年至2019年CML发病率（IRs）和伤残调整生命年（DALYs）数据。从ClinicalTrials.gov获取研究TKI用于CML的临床试验信息。对符合条件试验（N=30）的研究地点数据及DALYs进行分析，并按SDI水平汇总TKI批准情况、DALYs及发病率数据。 结果：1999年至2004年间，北美地区（NA）的DALYs和发病率年百分比变化（APC）显著下降。欧洲与中亚地区（ECA）和北美的发病率最高，而南亚地区（SAsia）和撒哈拉以南非洲地区（SSA）的DALYs负担最重。高SDI五分位国家的DALYs普遍低于低SDI国家。在TKI临床试验中，南亚、撒哈拉以南非洲及欧洲与中亚地区的DALYs负担与试验地点分布存在显著差异。所有30项试验均包含高SDI国家，且高SDI国家（142项）的TKI批准数量显著多于低SDI国家（14项）。 结论：临床试验的设计与招募阶段应加强对疾病负担较重国家的纳入，并提高其TKI药物可及性。当前代表性不足的现象凸显了全球医疗资源分配的不均衡问题。
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