title: Patient-Reported Outcomes in Chronic Heart Failure: Applicability for Regulatory Approval.
abstract: 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.
  
  
  
  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.
  
  
  
  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.
  
  
  
  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.
  
  
  
  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.
authors.affiliation: School of Medicine, University of California San Francisco and Section of Cardiology, San Francisco Veterans Affairs Medical Center, San Francisco, California.
authors.forename: Mitchell A
authors.initials: MA
authors.lastname: Psotka
authors.affiliation: Evidera, London, United Kingdom.
authors.forename: Robyn
authors.initials: R
authors.lastname: von Maltzahn
authors.affiliation: Evidera, Bethesda, Maryland.
authors.forename: Milena
authors.initials: M
authors.lastname: Anatchkova
authors.affiliation: Amgen Inc., Thousand Oaks, California.
authors.forename: Irene
authors.initials: I
authors.lastname: Agodoa
authors.affiliation: Amgen Inc., Thousand Oaks, California.
authors.forename: Dina
authors.initials: D
authors.lastname: Chau
authors.affiliation: Cytokinetics, Inc., South San Francisco, California.
authors.forename: Fady I
authors.initials: FI
authors.lastname: Malik
authors.affiliation: Department of Health Services, University of Washington, Seattle, Washington.
authors.forename: Donald L
authors.initials: DL
authors.lastname: Patrick
authors.affiliation: Saint Luke's Mid America Heart Institute and Department of Biomedical and Health Informatics, University of Missouri, Kansas City, Missouri.
authors.forename: John A
authors.initials: JA
authors.lastname: Spertus
authors.affiliation: Evidera, London, United Kingdom.
authors.forename: Ingela
authors.initials: I
authors.lastname: Wiklund
authors.affiliation: School of Medicine, University of California San Francisco and Section of Cardiology, San Francisco Veterans Affairs Medical Center, San Francisco, California. Electronic address: john.teerlink@ucsf.edu.
authors.forename: John R
authors.initials: JR
authors.lastname: Teerlink
mesh_terms: D002908:Chronic Disease; D018795:Device Approval; D017277:Drug Approval; D004348:Drug Labeling; D006333:Heart Failure / Q000503:physiopathology / Q000628:therapy*; D006801:Humans; D000071066:Patient Reported Outcome Measures; D011356:Product Labeling; D015203:Reproducibility of Results; D014481:United States; D014486:United States Food and Drug Administration
keywords: European Medicines Agency; Food and Drug Administration; chronic heart failure; clinical management; patient-reported outcomes; regulatory approval