Gupta Medhavi, Akhtar Othman Salim, Bahl Bhavyaa, Mier-Hicks Angel, Attwood Kristopher, Catalfamo Kayla, Gyawali Bishal, Torka Pallawi
Program in Women's Oncology, Brown University, Providence, Rhode Island, USA.
Medical College of Wisconsin, Milwaukee, Wisconsin, USA.
BMJ Oncol. 2024 Jul 11;3(1):e000369. doi: 10.1136/bmjonc-2024-000369. eCollection 2024.
Health-related quality of life (HRQoL) outcomes are important in making clinical and policy decisions. This study aimed to examine the HRQoL reporting in cancer drug trials leading to Food and Drug Administration (FDA) approvals.
This retrospective cohort study analysed HRQoL data for trials leading to FDA approvals between July 2015 and May 2020. Proportion of included trials that reported HRQoL, latency between FDA approval and first report of HRQoL data, HRQoL outcomes, and their correlation with OS (overall survival) and PFS (progression-free survival) were analysed.
Of the 233 trials associated with 207 FDA approvals, HRQoL was reported in 50% of trials, of which only 42% had the data reported by the time of FDA approval. There were no changes in frequency of HRQoL reporting between 2015 and 2020. HRQoL data were first reported in the primary publication in only 30% trials. Of the 115 trials with HRQoL data available, HRQoL improved in 43%, remained stable in 53% and worsened in 4% of trials. Among the trials that led to FDA approvals based on surrogate endpoints (79%), HRQoL was reported in 45% and improved only in 18% trials. There was no association between OS and PFS benefit and HRQoL outcomes.
Rates of HRQoL reporting were suboptimal in trials that led to FDA approvals with no improvements seen between 2015 and 2020. HRQoL reporting was often delayed and not presented in the primary publication. HRQoL reporting was further sparse in trials with approvals based on surrogate endpoints and HRQoL improved in only a minority of them.
与健康相关的生活质量（HRQoL）结果在临床和政策决策中很重要。本研究旨在检查导致美国食品药品监督管理局（FDA）批准的癌症药物试验中的HRQoL报告情况。
这项回顾性队列研究分析了2015年7月至2020年5月期间导致FDA批准的试验的HRQoL数据。分析了纳入试验中报告HRQoL的比例、FDA批准与HRQoL数据首次报告之间的间隔时间、HRQoL结果及其与总生存期（OS）和无进展生存期（PFS）的相关性。
在与207项FDA批准相关的233项试验中，50%的试验报告了HRQoL，其中只有42%在FDA批准时报告了数据。2015年至2020年期间，HRQoL报告的频率没有变化。HRQoL数据仅在30%的试验的主要出版物中首次报告。在有HRQoL数据的115项试验中，43%的试验中HRQoL有所改善，53%保持稳定，4%恶化。在基于替代终点获得FDA批准的试验中（79%），45%报告了HRQoL，只有18%的试验中HRQoL有所改善。OS和PFS获益与HRQoL结果之间没有关联。
在导致FDA批准的试验中，HRQoL报告率不理想，2015年至2020年期间未见改善。HRQoL报告经常延迟，且未在主要出版物中呈现。在基于替代终点获得批准的试验中，HRQoL报告更为稀少，且只有少数试验中HRQoL有所改善。
