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Review
. 2025 Apr 15;151(15):1111-1125.
doi: 10.1161/CIRCULATIONAHA.124.072158. Epub 2025 Apr 14.

Patient-Reported Outcomes as End Points in Heart Failure Trials

Javed Butler  1   2 , Muhammad Shariq Usman  3   4 , Charu Gandotra  5 , Ali Salman  6 , Andrew Farb  7 , Aliza M Thompson  5 , Norman Stockbridge  5 , Cordula Zeller  8 , Folke Folkvaljon  9
Affiliations
Review

Patient-Reported Outcomes as End Points in Heart Failure Trials

Javed Butler et al. Circulation. .

Abstract

Heart failure is a growing health-care concern affecting tens of millions of individuals globally. Although traditional therapeutic strategies have focused on reducing the risk for hospitalization and mortality, the importance of patient-reported outcomes (PROs) in patients with heart failure is increasingly being recognized. Regulatory agencies consider PROs part of their evaluation of drugs and devices, and professional society guidelines may recommend interventions that improve PROs. However, for several reasons, the effect of interventions on PROs reported in heart failure trials currently is difficult to interpret. There is no consensus on the timing and frequency of PRO assessments. Moreover, it has been difficult to establish a minimal clinically important difference, that is, the minimal change in a PRO score that is meaningful to a patient. In addition, traditional methods of analyzing and reporting PROs such as comparison of mean differences across groups or responder analysis are prone to statistical artifacts and misinterpretation. This article presents an in-depth discussion of these issues, with the Kansas City Cardiomyopathy Questionnaire used as an example, to facilitate the use of PROs in heart failure research, regulatory, and clinical settings.

Keywords: United States Food and Drug Administration; health status; heart failure; patient reported outcome measures; quality of life.

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Conflict of interest statement

Dr Butler consults to Abbott, American Regent, Amgen, Applied Therapeutic, AskBio, Astellas, AstraZeneca, Bayer, Boehringer Ingelheim, Boston Scientific, Bristol Myers Squibb, Cardiac Dimension, Cardiocell, Cardior, CSL Bearing, CVRx, Cytokinetics, Daxor, Edwards, Element Science, Faraday, Foundry, G3P, Innolife, Impulse Dynamics, Imbria, Inventiva, Ionis, Levator, Lexicon, Lilly, LivaNova, Janssen, Medtronics, Merck, Occlutech, Owkin, Novartis, Novo Nordisk, Pfizer, Pharmacosmos, Pharmain, Prolaio, Pulnovo, Regeneron, Renibus, Roche, Salamandra, Salubris, Sanofi, SC Pharma, Secretome, Sequana, SQ Innovation, Tenex, Tricog, Ultromics, Vifor, and Zoll. Drs Usman and Salman report no conflicts. Drs Gandotra, Thompson, and Stockbridge are employees of the FDA, USA. C. Zeller is employed by Boehringer Ingelheim Pharma GmbH & Co KG, but the current work was undertaken independently; this work was not sponsored by Boehringer Ingelheim, and the views in this manuscript may not represent the views of Boehringer Ingelheim. F. Folkvaljon is employed by AstraZeneca, but the current work was undertaken independently; this work was not sponsored by AstraZeneca, and the views in this manuscript may not represent the views of AstraZeneca. All authors met criteria for authorship as recommended by the International Committee of Medical Journal Editors (ICMJE). The authors did not receive payment related to the development of the paper.

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