PO.RSP01.01 · 监管科学与政策
非小细胞肺癌早期临床试验中心在美国境内外的分布
The distribution of non-small cell lung cancer early phase clinical trial sites within and outside the United States
作者与单位 Authors & Affiliations
摘要 Abstract
中文摘要
近期监管指南概述了将临床试验纳入常规诊疗并使其更贴近美国患者居住地的相关考量，凸显了美国食品药品监督管理局（FDA）对试验数据适用于真实世界患者人群的重视。然而，近期肿瘤药物顾问委员会（Oncologic Drugs Advisory Committee）会议表明，美国患者入组不足在肿瘤学试验中仍是一个问题，令人质疑试验结果与美国患者人群的相关性和代表性。除了试验入组和完成时间过长等因素外，试验中心饱和一直被坊间引为将试验推向美国境外的一个因素。为了解临床试验中心地点的近期趋势，LUNGevity Foundation 以肺癌为案例，评估了早期临床试验中心随时间推移在美国境内外的分布。这项定量描述性评估利用 ClinicalTrials.gov 数据，分析了 2020 年 1 月至 2024 年 12 月开始的、针对非小细胞肺癌（NSCLC）的早期 I 期或 I 期干预性、企业申办的临床试验。唯一中心（unique site）定义为至少有一项试验（以唯一 NCT 编号标识）所在的单一地址。试验实例（trial instance）定义为一项唯一试验在一个唯一中心的启动。总体而言，在研究期内共开展了 555 项试验，覆盖 47 个国家所有中心，共计 8,393 个试验实例。试验中心主要位于美国（45%）、中国（11%）、西班牙（8%）、韩国（5%）、法国（4%）和澳大利亚（4%）。虽然开展试验的数量在 2024 年之前持续增加，但无论是全球还是美国境内，唯一试验中心和试验实例的数量均自 2022 年开始下降，并持续下降至 2024 年。值得注意的是，在整个研究期内，至少有一项试验开展的美国中心数量显著减少（从 2020 年的 395 个减少至 2024 年的 223 个）。然而，试验量最高的中心（前 20 个中心在研究期内至少开展了 27 项试验）并未随时间出现这种下降。该分析显示出一种令人担忧的趋势，即试验向美国表现最佳的中心集中，进一步印证了对中心饱和的担忧，并与去中心化、将试验推向社区的呼吁相冲突。尚需进一步研究以了解这些趋势是否在后期临床试验及其他肿瘤类型中持续存在。
查看英文原文 English abstract
Recent regulatory guidance outlining considerations for bringing clinical trials into routine care and closer to where US patients live highlights the importance the U.S. Food and Drug Administration places on the applicability of trial data to real-world patient populations. However, recent Oncologic Drugs Advisory Committee meetings reveal that insufficient enrollment of US patients remains an issue in oncology trials, calling into question the relevance and representativeness of trial results to the US patient population. In addition to factors like long timelines to trial accrual and completion, trial site saturation has been anecdotally cited as a factor pushing trials outside of the US. To understand recent trends in the locations of clinical trial sites, LUNGevity Foundation used lung cancer as a use case to assess the distribution of early phase clinical trial sites within and outside of the US over time. This quantitative descriptive assessment utilized ClinicalTrials.gov data to analyze interventional, industry-sponsored clinical trials for non-small cell lung cancer (NSCLC) in early phase I or phase I opening in January 2020-December 2024. Unique sites were defined as a single address where at least one trial, identified by a unique NCT number, was located. Trial instances were defined as the initiation of a unique trial at a unique site. Overall, 555 trials opened within the study period for a total of 8,393 trial instances across all sites in 47 countries. Trial sites were largely located in the US (45%), China (11%), Spain (8%), Korea (5%), France (4%), and Australia (4%). While the number of trials opening increased until 2024, the number of unique trial sites and trial instances, both globally and within the US, started decreasing in 2022 and continued to decrease through 2024. Notably, the number of US sites with at least one trial opening markedly decreased during the entire study period (395 sites in 2020 to 223 sites in 2024). However, the sites with the highest trial volume (top 20 sites had at least 27 trials open in the study period) did not see this decrease over time. This analysis demonstrates a concerning trend towards trial consolidation at top performing sites in the US, further supporting concerns regarding site saturation and conflicting with appeals to decentralize and move trials into the community. Further work is needed to understand if these trends persist in later phase clinical trials and other tumor types.
利益披露 Disclosure
B. A. McKelvey, None..
L. Saxton-Stivers, None..
T. Guidry, None.