Patient Familiarity with, Understanding of, and Preferences for Clinical Trial Endpoints and Terminology

Keywords

clinical trial, endpoints, breast cancer, quality of life, outcomes, patient-reported outcomes, comprehension

Abstract

Background: While there is increased attention to designing and explaining clinical trials in ways that are clinically meaningful for patients, there is limited information on patient preferences, understanding and perceptions of this content.

Methods: Using maximum difference scaling (MaxDiff) methodology, we developed a survey assessing understanding of 19 clinical terms and perceived importance of nine endpoint surrogate phrases employed in clinical trials and consent forms. The survey was administered electronically to individuals with metastatic breast cancer affiliated with the Metastatic Breast Cancer Alliance. Analysis was performed using the “bayesm” package in the R Statistical Package.

Results: Among 503 respondents, 77% had a college degree, 70% were diagnosed with metastatic disease ≥2 years prior to survey completion, and 77% had received ≥2 lines of systemic therapy. Less than 35% of respondents reported understanding “fairly well” the terms “symptomatic progression,” “duration of disease control,” “time to treatment cessation,” and “endpoints.” Income level and time since onset of metastatic disease correlated with comprehension. Patients who had received six or more lines of therapy perceived “time until serious side effects” (P< 0.001) and “time on therapy” (P< 0.001) to be more important compared to patients who had received one line of therapy. Positively phrased parameters were associated with increased perceived importance.

Conclusions: Even among educated, heavily pre-treated patients, many commonly used clinical research terms are poorly understood. Comprehension and perceived importance of trial endpoints varies over the course of disease. These observations may inform the design, discussion, and reporting of clinical trials.

Original Publication Citation

Mitchell S. von Itzstein, Elda Railey, Mary L. Smith, Carol B. White, George W. Sledge Jr., John R. Howell, Wendy Lawton, Donna M. Marinucci, Nisha Unni, and David E. Gerber, "Patient familiarity with, understanding of, and preferences for clinical trial endpoints and terminology", Cancer, 2020.

Document Type

Peer-Reviewed Article

Publication Date

2020

Publisher

Cancer

Language

English

College

Marriott School of Business

Department

Marketing

University Standing at Time of Publication

Assistant Professor

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