Expanding the use and interpretation of patient-centric cardiovascular clinical trial endpoints.

Abstract

Significant improvements have been achieved to enhance the patient-centricity of clinical research, including the development and utilization of novel clinical trial endpoints. These include endpoints that harness outcomes that are important to patients and reflect the patients' lived experiences. This may take the form of utilizing variables such as days alive and out of hospital (DAOH) and quality-of-life adjusted outcomes. The use of composite outcomes can be used to enrich patient-centricity by weighting or ranking events. These approaches have several nuances that should be considered including selecting appropriate events, defining outcomes, how to elicit or construct weights, and whose opinions to consider. After weights have been determined, a variety of approaches exist to combine weights with outcomes and make comparisons between groups. The approaches, including the win ratio, weighted win ratio, desirability of outcome ranking (DOOR), multicriteria decision analysis (MCDA), and variations of time-to-first composite event analyses, have unique advantages and challenges depending on the clinical scenario. While improving patient-centric outcomes is of high importance to multiple stakeholders, more comparative work is needed to characterize the implications of alternative approaches.