Author + information
- Received January 25, 2016
- Revision received March 14, 2016
- Accepted April 27, 2016
- Published online October 1, 2016.
- Mitchell A. Psotka, MD, PhDa,
- Robyn von Maltzahn, PhDb,
- Milena Anatchkova, PhDc,
- Irene Agodoa, MDd,
- Dina Chau, PharmDd,
- Fady I. Malik, MD, PhDe,
- Donald L. Patrick, MPH, PhDf,
- John A. Spertus, MPH, MDg,
- Ingela Wiklund, PhDb and
- John R. Teerlink, MDa,∗ ()
- aSchool of Medicine, University of California San Francisco and Section of Cardiology, San Francisco Veterans Affairs Medical Center, San Francisco, California
- bEvidera, London, United Kingdom
- cEvidera, Bethesda, Maryland
- dAmgen Inc., Thousand Oaks, California
- eCytokinetics, Inc., South San Francisco, California
- fDepartment of Health Services, University of Washington, Seattle, Washington
- gSaint Luke’s Mid America Heart Institute and Department of Biomedical and Health Informatics, University of Missouri, Kansas City, Missouri
- ↵∗Reprint requests and correspondence:
Dr. John R. Teerlink, San Francisco VA Medical Center, Cardiology, 111C; Building 203, Room 2A-49, 4150 Clement Street, San Francisco, California 94121-1545.
Objectives The study sought to review the characteristics of existing patient-reported outcome (PRO) instruments used with chronic heart failure (HF) patients and evaluate their potential to support an approved U.S. Food and Drug Administration (FDA) product label claim.
Background PROs, including symptoms and their associated functional limitations, contribute substantially to HF patient morbidity. PRO measurements capture the patient perspective and can be systematically assessed with structured questionnaires, however rigorous recommendations have been set by the FDA regarding the acceptability of PRO measures as a basis for product label claims.
Methods Extensive searches of databases and specialty guidelines identified PRO instruments used in patients with chronic HF. Information on critical properties recommended by the FDA guidance were systematically extracted and used to evaluate the selected PRO instruments.
Results Nineteen PRO instruments used with chronic HF patients were identified. The Kansas City Cardiomyopathy Questionnaire and Minnesota Living with Heart Failure Questionnaire were the most extensively evaluated and validated in studies of this population. However, judged by criteria listed in the FDA PRO guidance, no existing PRO measure met all of the criteria to support a product label claim in the United States.
Conclusions Currently available chronic HF PRO measures do not fulfill all the recommendations provided in the FDA PRO guidance and therefore may not support an FDA-approved product label claim. Future investigations are merited to develop a PRO measure for use in patients with chronic HF in accordance with the FDA guidance.
- chronic heart failure
- clinical management
- European Medicines Agency
- Food and Drug Administration
- patient-reported outcomes
- regulatory approval
This study was sponsored by Amgen Inc. Dr. von Maltzahn is a former employee and Drs. Anatchkova and Wiklund are current employees of Evidera, which received funding from Amgen Inc. to conduct this study. Dr. Agodoa is a former employee and shareholder of Amgen, Inc. Dr. Chau is a current employee and shareholder of Amgen Inc. Dr. Malik is an employee and shareholder of Cytokinetics, Inc., which has collaborated with Amgen Inc. in the development of omecamtiv mecarbil. Dr. Patrick is a consultant to Amgen Inc. Dr. Spertus served as a consultant to Amgen Inc. for this study; is a consultant to Janssen, Amgen Inc., Novartis Pharmaceuticals Corporation, Bayer Healthcare, and Regeneron Pharmaceuticals, Inc.; has received grant support from Gilead Sciences, Inc., Eli Lilly and Co., the National Institutes of Health, American Heart Association, the Patient-Centered Outcomes Research Institute, and the Aetna Foundation; has equity interest in Health Outcomes Sciences; and owns the copyright to the Seattle Angina Questionnaire, Peripheral Artery Questionnaire, and Kansas City Cardiomyopathy Questionnaire. Dr. Teerlink has received grant/research support and is a consultant to Amgen Inc., Bayer, Cytokinetics, Inc., Novartis Pharmaceuticals Corp., Relypsa, and Trevena Inc. All other authors have reported that they have no relationships relevant to the contents of this paper to disclose.
- Received January 25, 2016.
- Revision received March 14, 2016.
- Accepted April 27, 2016.
- 2016 American College of Cardiology Foundation