In oncology, phase II or multiple expansion cohort trials are crucial for clinical development plans. This is because they aid in identifying potent agents with sufficient activity to continue development and confirm the proof of concept. Typically, these clinical trials are single-arm trials, with the primary endpoint being short-term treatment efficacy. Despite the development of several well-designed methodologies, there may be a practical impediment in that the endpoints may be observed within a sufficient time such that adaptive go/no-go decisions can be made in a timely manner at each interim monitoring. Specifically, Response Evaluation Criteria in Solid Tumors guideline defines a confirmed response and necessitates it in non-randomized trials, where the response is the primary endpoint. However, obtaining the confirmed outcome from all participants entered at interim monitoring may be time-consuming as non-responders should be followed up until the disease progresses. Thus, this study proposed an approach to accelerate the decision-making process that incorporated the outcome without confirmation by discounting its contribution to the decision-making framework using the generalized Bayes' theorem. Further, the behavior of the proposed approach was evaluated through a simple simulation study. The results demonstrated that the proposed approach made appropriate interim go/no-go decisions.

翻译：在肿瘤学中，二期或多项扩展队列试验对于临床开发计划至关重要。这是因为它们有助于识别具有足够活性、可继续开发并确认概念验证的有效药物。通常，这些临床试验为单臂试验，主要终点为短期治疗效果。尽管已开发出多种设计良好的方法，但实践中可能存在一个障碍：终点需在足够时间内被观察到，以便在每次期中监测时及时做出适应性继续/停止决策。具体而言，《实体瘤疗效评价标准》指南定义了确认缓解，并在以缓解为主要终点的非随机试验中要求进行确认。然而，从所有在期中监测时入组的参与者处获得确认结果可能耗时较长，因为对无缓解者需随访至疾病进展。因此，本研究提出了一种加速决策过程的方法，该方法通过广义贝叶斯定理对未确认结果的贡献进行折减，将其纳入决策框架。此外，通过简单的模拟研究评估了所提方法的表现。结果表明，所提方法能够做出恰当的期中继续/停止决策。