SWOG Statistics and Data Management Center, Seattle, Washington.
Fred Hutchinson Cancer Research Center, Seattle, Washington.
JAMA Netw Open. 2019 Sep 4;2(9):e1910593. doi: 10.1001/jamanetworkopen.2019.10593.
National Cancer Institute Clinical Trial Network (NCTN) groups serve a vital role in identifying effective new antineoplastic regimens. However, the downstream clinical effect of their trials has not been systematically examined.
To examine the association of NCTN trials with guideline care and new drug indications.
DESIGN, SETTING, AND PARTICIPANTS: This retrospective cohort study evaluated phase 3 SWOG Cancer Research Network clinical trials from January 1, 1980, through June 30, 2017. Only completed trials with published results were included. To be considered practice influential (PI), a trial must have been associated with guideline care through its inclusion in National Comprehensive Cancer Network (NCCN) clinical guidelines or US Food and Drug Administration (FDA) new drug approvals in favor of a recommended treatment. Data were analyzed from June 15, 2018, through March 29, 2019.
Estimated overall rate of PI trials, as well as trends over time. The total federal investment supporting the set of trials was also determined.
In total, 182 trials consisting of 148 028 patients were studied. Eighty-two studies (45.1%; 95% CI, 37.7%-52.6%) were PI, of which 70 (38.5%) influenced NCCN guidelines, 6 (3.3%) influenced FDA new drug approvals, and 6 (3.3%) influenced both. The number of PI trials was 47 of 65 (72.3%) among those with positive findings and 35 of 117 (29.9%) among those with negative findings. Thus, 35 of 82 PI trials (42.7%) were based on studies with negative findings, with nearly half of these studies (17 of 35 [48.6%]) reaffirming standard of care compared with experimental therapy. The total federal investment spent in conducting the trials was $1.36 billion (2017 US dollars), a rate of $7.5 million per study or $16.6 million per PI trial.
Nearly half of all phase 3 trials by one of the NCTN's largest groups were associated with guideline care or new drug indications, including those with positive and negative findings. Compared with the costs of a new drug approval in pharmaceutical companies, typically estimated at more than $1 billion, the amount of federal funds invested to provide this valuable evidence was modest. These results suggest that the NCTN program contributes clinically meaningful, cost-efficient evidence to guide patient care.
美国国家癌症研究所临床试验网络（NCTN）小组在确定有效的新抗肿瘤方案方面发挥着至关重要的作用。然而，其试验的下游临床效果尚未得到系统评估。
研究 NCTN 试验与指南护理和新药适应证之间的关联。
设计、地点和参与者：本回顾性队列研究评估了 1980 年 1 月 1 日至 2017 年 6 月 30 日期间进行的 SWOG 癌症研究网络的 III 期临床试验。仅纳入了有已发表结果的已完成试验。如果一项试验因其纳入了国家综合癌症网络（NCCN）临床指南或美国食品药品监督管理局（FDA）的新药批准而与推荐的治疗方法一致，则被认为对实践有影响（PI）。数据于 2018 年 6 月 15 日至 2019 年 3 月 29 日进行分析。
估计 PI 试验的总体比例，以及随时间的趋势。还确定了支持这组试验的联邦总投资。
共纳入了 182 项临床试验，包括 148028 名患者。82 项研究（45.1%；95%CI，37.7%-52.6%）为 PI，其中 70 项（38.5%）影响了 NCCN 指南，6 项（3.3%）影响了 FDA 新药批准，6 项（3.3%）同时影响了这两者。阳性发现的试验中有 47 项（72.3%）为 PI 试验，阴性发现的试验中有 35 项（29.9%）为 PI 试验。因此，82 项 PI 试验中有 35 项（42.7%）基于阴性发现的研究，其中近一半（17 项）与实验治疗相比，再次证实了标准护理。进行这些试验的联邦总投资为 13.6 亿美元（2017 年美元），即每项研究 750 万美元或每项 PI 试验 1660 万美元。
美国 NCTN 最大的研究小组之一进行的 III 期试验中，近一半与指南护理或新药适应证相关，包括阳性和阴性发现的试验。与制药公司新药批准的成本相比，通常估计超过 10 亿美元，联邦为提供这些有价值证据而投入的资金数额适中。这些结果表明，NCTN 计划为指导患者护理提供了具有临床意义、成本效益的证据。
