肿瘤学中的一些治疗策略问题可以通过使用观察数据的研究来回答。目标试验模拟是将随机对照试验的设计原理应用于观察数据分析，以减少设计引起的偏差。我们的目标是确定医生更愿意计划哪种类型的研究来回答缺乏肿瘤学证据的比较有效性问题。我们在肿瘤学专业医生中发起了一项在线调查。我们构建了一个基于小插图的查询，其中小插图描述了可以用来回答预定义问题的研究场景。我们设计了六个小插图，通过研究设计（随机对照试验或带有试验模拟框架的观察性研究）、主要研究特征、研究成功的概率以及结果可用之前的预期延迟来描述。参与者随机评估了五个小插图的成对比较，并被询问他们更愿意使用李克特量表来计划哪项研究。主要结果是评估临床医生对每次配对比较的偏好。计算了偏好得分的平均值和中位数。213 名专门研究多种肿瘤类型的参与者评估了至少一项比较，其中 82% 的参与者报告法国为其所属国家。两两比较的四分位数范围为 -4 到 4。在五次比较中，中位偏好评分不利于单中心随机对照试验。与单中心模拟试验 4 相比，中位偏好评分强烈支持多中心国家模拟试验 [IQ 2.5-4]。大型欧洲观察性研究的平均偏好得分最高，为 1.14 (SD 3.33)，而单中心随机对照试验的平均偏好得分最低 -1.86 (SD 2.93)。没有强烈偏好的研究设计，但单中心随机对照试验的平均偏好得分最低。随机对照试验是成对比较中最不受欢迎的研究。新研究的计划是科学合理性、可行性、成本和获得结果之前的时间之间的折衷。我们需要在正确的时间对正确的问题提供正确的答案。版权所有 © 2024。由 Elsevier Inc. 出版。

Some therapeutic strategy questions in oncology could be answered with studies using observational data. Target trial emulation is the application of design principles from randomized controlled trials to the analysis of observational data, to reduce design-induced biases. Our objective was to determine which type of study physicians would preferably plan to answer a comparative effectiveness question lacking evidence in oncology.We launched an online survey among physicians specialized in oncology. We constructed a vignette-based inquiry where vignettes described study scenarios which could be conducted to answer the predefined question. We designed six vignettes described by study design (randomized controlled trial or observational study with a trial emulation framework), main study characteristics, probability of the study succeeding and anticipated delay before results availability. Participants randomly assessed five pair-wise comparisons of the vignettes and were asked which study they would preferably plan by using a Likert scale. The main outcome was the evaluation of clinicians' preferences for each pairwise comparison. Mean and median preference scores were calculated.213 participants, specialized in many tumor types, assessed at least one comparison with 82% reporting France as their country of affiliation. The interquartile range was -4 to 4 across pairwise comparisons. The median preference score was in disfavor of the monocentric randomized controlled trial for the five comparisons where it appeared. The median preference score was strongly in favor of the multicentric national emulated trial when compared to the monocentric emulated trial 4 [IQ 2.5-4]. The mean preference score was the highest for the large European observational study 1.14 (SD 3.33), while the mean preference score was the lowest for the monocentric randomized controlled trial -1.86 (SD 2.93).No study design was strongly preferred, but the monocentric randomized controlled trial was the least favored study in pair-wise comparisons. The planification of the new research is a compromise between scientific soundness, feasibility, cost, and time before obtaining results. We need to have the right answers to the right questions at the right time.Copyright © 2024. Published by Elsevier Inc.