Cedars-Sinai Medical Center, Los Angeles, CA.
SWOG Statistics and Data Management Center, Seattle, WA.
Am Soc Clin Oncol Educ Book. 2024 Jun;44(3):e100040. doi: 10.1200/EDBK_100040.
Clinical trials are essential for advancing oncology treatment strategies and have contributed significantly to the decline in cancer mortality rates over the past decades. Traditional explanatory trials, focused on establishing intervention efficacy in ideal settings, often lack generalizability and may not reflect real-world patient care scenarios. Furthermore, increasing complexity in cancer clinical trial design has led to challenges such as protocol deviations, slow enrollment leading to lengthened durations of trial, and escalating costs. By contrast, pragmatic trials aim to assess intervention effectiveness in more representative patient populations under routine clinical conditions. Here, we review the principles, methodologies, challenges, and advantages of incorporating pragmatic features (PFs) into cancer clinical trials. We illustrate the application of pragmatic trial designs in oncology and discuss the QUASAR collaborative, TAPUR study, and the ongoing PRAGMATICA-LUNG trial. Although not all oncology trials may be amenable to adopting fully pragmatic designs, integration of PFs when feasible will enhance trial generalizability and real-world applicability. Project Pragmatica and similar initiatives advocate for the integration of real-world practice with clinical trials, fostering a nuanced approach to oncology research that balances efficacy and effectiveness assessments, ultimately with a goal of improving patient outcomes.
临床研究对于推进肿瘤治疗策略至关重要，在过去几十年中，癌症死亡率的下降在很大程度上归功于临床研究。传统的解释性试验侧重于在理想环境下确定干预措施的疗效，往往缺乏普遍性，可能无法反映真实的患者护理场景。此外，癌症临床试验设计的日益复杂带来了一些挑战，如方案偏离、入组缓慢导致试验持续时间延长，以及成本上升。相比之下，实用临床试验旨在评估常规临床条件下更具代表性的患者群体中干预措施的效果。在这里，我们回顾了将实用特征（PFs）纳入癌症临床试验的原则、方法、挑战和优势。我们举例说明了实用试验设计在肿瘤学中的应用，并讨论了 QUASAR 协作研究、TAPUR 研究以及正在进行的 PRAGMATICA-LUNG 试验。虽然并非所有肿瘤学试验都适合采用完全实用的设计，但在可行的情况下纳入 PFs 将增强试验的普遍性和现实适用性。Project Pragmatica 和类似的倡议主张将真实世界的实践与临床试验相结合，为肿瘤学研究提供一种细致的方法，平衡疗效和有效性评估，最终目标是改善患者的预后。
