Gerber David E, Wynters Claire R, Prasad Tanushree, Schnel Ronny K, Zhang Song, Stinchcombe Thomas E, Villaruz Liza C, Bauml Joshua M, Iams Wade T, Patil Tejas, Liu Stephen V, Horn Leora, Hudak John M, Camidge D Ross
Harold C. Simmons Comprehensive Cancer Center, UT Southwestern Medical Center, Dallas, Texas.
Criterium, Inc., Saratoga Springs, New York.
Clin Cancer Res. 2025 Mar 17;31(6):1103-1108. doi: 10.1158/1078-0432.CCR-24-3460.
Investigator-initiated trials (IIT) may address important biological and clinical questions that may not be prioritized by pharmaceutical sponsors. However, little is known about the process by which IIT proposals are evaluated and activated.
We performed a retrospective study of IIT concepts submitted through the Academic Thoracic Oncology Medical Investigators Consortium, which comprises 13 institutions in the United States and Canada, from consortium inception in 2014 to 2024. We compared approved and disapproved concepts using χ2 tests, Fisher exact tests, and Wilcoxon rank-sum tests.
Among 68 presented IIT concepts, 60 (88%) received consortium approval a median of 30 days (IQR, 31-59 days) after submission. Concepts submitted by junior faculty were more likely to be approved than those from full professors (P = 0.003). Of the 60 concepts subsequently submitted to pharmaceutical sponsors, 15 (25%) were approved, 43 (72%) were disapproved, and 2 (3%) remain under review. The median time between concept submission to a sponsor and the sponsor's decision was 61 days (IQR, 31-183 days). Concepts with shorter projected durations were more likely to be approved by the pharmaceutical sponsor (P = 0.05). For sponsor-approved IIT concepts, the median overall time from initial submission to trial activation was 18 months.
Only a small proportion of proposed investigator-initiated cancer clinical trials are successfully activated following a prolonged development process. Given the importance of IITs in addressing real-world, practical questions and the growing professional challenges facing clinical research physician faculty, further attention to IIT development facilitators and barriers is warranted.
研究者发起的试验（IIT）可能会解决制药赞助商未优先考虑的重要生物学和临床问题。然而，对于IIT提案的评估和启动过程，我们知之甚少。
我们对通过学术胸部肿瘤医学研究者联盟提交的IIT概念进行了回顾性研究，该联盟由美国和加拿大的13个机构组成，研究时间从2014年联盟成立至2024年。我们使用卡方检验、Fisher精确检验和Wilcoxon秩和检验比较了获批和未获批的概念。
在提交的68个IIT概念中，60个（88%）在提交后中位30天（四分位间距，31 - 59天）获得联盟批准。初级教员提交的概念比正教授提交的更有可能获得批准（P = 0.003）。在随后提交给制药赞助商的60个概念中，15个（25%）获得批准，43个（72%）未获批，2个（3%）仍在审核中。从向赞助商提交概念到赞助商做出决定的中位时间为61天（四分位间距，31 - 183天）。预计持续时间较短的概念更有可能获得制药赞助商的批准（P = 0.05）。对于赞助商批准的IIT概念，从最初提交到试验启动的中位总时间为18个月。
在漫长的开发过程之后，只有一小部分提议的研究者发起的癌症临床试验成功启动。鉴于IIT在解决实际问题方面的重要性以及临床研究医师教员面临的日益增加的专业挑战，有必要进一步关注IIT开发的促进因素和障碍。
