Minimal clinically important improvement/difference (MCII/MCID) and patient acceptable symptom state (PASS): what do...

Area of Science:

• Clinical Trials Methodology
• Patient-Reported Outcomes
• Health Measurement

Background:

• Growing emphasis on patient-centered endpoints in clinical research.
• Need for standardized methods to define meaningful clinical improvement.
• Categorical endpoints like Minimal Clinically Important Improvement (MCII) and Patient Acceptable Symptom State (PASS) are increasingly utilized.

Purpose of the Study:

• To outline the definitions and statistical approaches for establishing cut-points for MCII and PASS.
• To demonstrate how these patient-defined thresholds can be integrated into clinical trial endpoints.
• To provide a framework for interpreting clinical trial results based on patient-relevant outcomes.

Main Methods:

• Defining MCII as the smallest change signifying important improvement from the patient's perspective.
• Defining PASS as the highest symptom level beyond which patients feel well.
• Utilizing the 75th percentile method and Receiver Operating Characteristic (ROC) curves to identify cut-points.

Main Results:

• Identified cut-points for MCII and PASS provide objective measures of clinically meaningful change.
• These cut-points can be incorporated as trial endpoints, quantifying patient-reported outcomes.
• Enables reporting on the proportion of patients achieving meaningful improvement and acceptable symptom states.

Conclusions:

• MCII and PASS are valuable patient-centered endpoints for clinical trials.
• Statistical methods like the 75th percentile and ROC curves effectively determine relevant cut-points.
• Incorporating these endpoints enhances the clinical relevance and interpretability of trial findings.