A Clinical Instrument to Measure Patient Anecdotes in Clinical Trials

TL;DR

This paper introduces Clinical IMPACT, a new qualitative tool for capturing meaningful non-seizure treatment benefits in neurological trials, enhancing sensitivity and providing valuable insights into patient quality of life.

Contribution

The paper presents a novel instrument that quantifies patient anecdotes and ranks them for rigorous analysis, improving assessment of non-seizure outcomes in epilepsy trials.

Findings

Effective in capturing qualitative treatment benefits

Resistant to type 1 error in analysis

Enhances sensitivity of neurological trial outcomes

Abstract

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.