Clinical trials assessing neurological treatment are challenging due to the diversity of brain function, and the difficulty in quantifying it. Traditional treatment studies in epilepsy use seizure frequency as the primary outcome measure, which may overlooking meaningful improvements in patients' quality of life. This paper introduces the Clinical Instrument for Measuring Patient Anecdotes in Clinical Trials (Clinical IMPACT), a novel tool designed to capture qualitative non-seizure improvement across neurological domains. The Clinical IMPACT incorporates open-ended inquiries that allow participants or caregivers to identify and select anecdotal evidence of their most significant treatment benefits. A blinded panel of experts ranks these anecdotes, facilitating a rigorous statistical analysis using the Wilcoxon Rank-Sum Test to detect treatment efficacy. The approach is resistant to type 1 error, yet comprehensive in its ability to capture real-world effects on quality of life. The potential of the Clinical IMPACT tool to enhance sensitivity while also providing qualitative insights that can inform patients, healthcare providers, and regulatory bodies about treatment effects makes it important to consider in any neurological trial. We describe how it can be used in epilepsy, and advocate for its inclusion as a key secondary endpoint to provide a perspective on non-seizure outcomes, which have previously been challenging to measure, let alone to interpret, even when the clinical trial is positive.

翻译：由于大脑功能的多样性及其量化的困难性，评估神经治疗效果的临床试验面临挑战。传统癫痫治疗研究以发作频率作为主要结局指标，这可能忽略了患者生活质量的显著改善。本文介绍了临床试验中测量患者轶事的临床工具（Clinical IMPACT），这是一种新颖的工具，旨在捕捉跨神经功能领域的定性非发作性改善。Clinical IMPACT 采用开放式询问，允许参与者或护理人员识别并选择其最显著治疗益处的轶事证据。由盲法专家小组对这些轶事进行排序，并利用Wilcoxon秩和检验进行严格的统计分析以检测治疗效果。该方法能有效控制I类错误，同时全面捕捉对生活质量的真实影响。Clinical IMPACT 工具在提高敏感性的同时，还能提供定性见解，帮助患者、医疗提供者和监管机构了解治疗效果，因此在任何神经学试验中都值得考虑。我们描述了其在癫痫研究中的应用，并主张将其作为关键次要终点纳入，以提供非发作结局的视角——这些结局以往难以测量，更难以解释，即使在临床试验结果呈阳性的情况下也是如此。