Internal Medicine Department, Kaiser Permanente - Oakland Medical Center, Oakland, CA.
Division of Hematological Malignancies and Cellular Therapeutics, University of Kansas, Kansas City, KS.
JCO Glob Oncol. 2022 Aug;8:e2200119. doi: 10.1200/GO.22.00119.
The globalization of clinical trials has accelerated recent advances in multiple myeloma (MM). However, it is unclear whether trial enrollment locations are reflective of the global burden of MM and whether access to novel therapies is timely and equitable for countries that participate in those trials.
To assess this, we characterized where MM trials that led to US Food and Drug Administration (FDA) approvals were conducted and determined how often and quickly these drug regimens received approval in their participating trial countries on the basis of country income level and geographic region.
A systematic review was conducted to identify all MM clinical trials that met their primary endpoint, enrolled patients outside the United States, and resulted in FDA approval from 2005 to 2019. A total of 18 pivotal MM clinical trials were identified. High-income countries enrolled patients in 100% (18/18) of the trials identified, whereas upper-middle and lower-middle-income countries were represented in 61% (11/18) and 28% (5/18) of trials, respectively. No patients from low-income countries were enrolled. One trial enrolled patients in sub-Saharan Africa, and no trials enrolled patients in South Asia/Caribbean. For drugs/regimens that were approved in their participating countries, the median time from FDA approval to approval was 10.9 months. There were no drugs approved in lower-middle-income trial countries. MM trials leading to FDA approval are generally run in high-income, European, and Central Asian countries.
There are substantial disparities in where novel therapies are evaluated and where they are ultimately approved for use on the basis of income level and geography.
临床试验的全球化加速了多发性骨髓瘤（MM）的近期进展。然而，尚不清楚试验参与地点是否反映了 MM 的全球负担，以及参与这些试验的国家是否能够及时公平地获得新疗法。
为了评估这一点，我们描述了导致美国食品和药物管理局（FDA）批准的 MM 试验的进行地点，并根据国家收入水平和地理位置确定这些药物方案在其参与试验的国家获得批准的频率和速度。
进行了一项系统评价，以确定所有符合主要终点、在美国境外招募患者并导致 FDA 批准的 MM 临床试验，时间范围为 2005 年至 2019 年。共确定了 18 项关键 MM 临床试验。高收入国家参与了 100%（18/18）确定的试验，而上中收入和中下收入国家分别占 61%（11/18）和 28%（5/18）的试验。没有低收入国家的患者参与。一项试验招募了撒哈拉以南非洲的患者，没有试验招募了南亚/加勒比地区的患者。对于在其参与国家获得批准的药物/方案，从 FDA 批准到批准的中位时间为 10.9 个月。中下收入试验国家没有批准任何药物。导致 FDA 批准的 MM 试验通常在高收入、欧洲和中亚国家进行。
根据收入水平和地理位置，新疗法的评估和最终批准存在很大差异。
