Patient-Reported Outcomes in Chronic Heart Failure: Applicability for Regulatory Approval

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• 作者：Mitchell A. Psotka ; MD ; PhD^a ; Robyn von Maltzahn ; PhD^b ; Milena Anatchkova ; PhD^c ; Irene Agodoa ; MD^d ; Dina Chau ; PharmD^d ; Fady I. Malik ; MD ; PhD^e ; Donald L. Patrick ; MPH ; PhD^f ; John A. Spertus ; MPH ; MD^g ; Ingela Wiklund ; PhD^b ; John R. Teerlink ; MD^a ; ^{john.teerlink@ucsf.edu" class="auth_mail" title="E-mail the corresponding author}
• 关键词：chronic heart failure ; clinical management ; European Medicines Agency ; Food and Drug Administration ; patient-reported outcomes ; regulatory approval
• 刊名：JACC: Heart Failure
• 出版年：2016
• 出版时间：October 2016
• 年：2016
• 卷：4
• 期：10
• 页码：791-804
• 全文大小：699 K

文摘

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.