Reasons for rejection of patient-reported outcome label claims: a compilation based on a review of patient-reported outcome use among new molecular entities and biologic license applications, 2006-2010

Value in Health : the Journal of the International Society for Pharmacoeconomics and Outcomes Research
Carla DeMuroAri Gnanasakthy

Abstract

Previous analyses of patient-reported outcome (PRO) label claims concentrated only on successful label claims. The goal of this research was to explore the reasons why PRO label claims were denied and to compile regulatory feedback regarding the use of PROs in clinical trials. By using the Food and Drug Administration's Drug Approval Report Web page, all new molecular entities and biologic license applications approved between January 2006 and December 2010 were identified. For identified drug products, medical review sections from publicly available drug approval packages were reviewed to identify PRO end-point status and any Study Endpoints and Label Development team comments. Of the 116 new molecular entities and biologic license applications with accompanying drug approval packages identified and reviewed, 44.8% of the products included PROs as part of the pivotal studies; however, only 24.1% received PRO label claims. Primary reasons for denial included issues of fit for purpose, issues of study design, data quality or interpretation, statistical issues, administrative issues, and lack of demonstrated treatment benefit. Based on drug approval packages, nearly half (45%) of new molecular entitity/biologic license applicatio...Continue Reading

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Aug 28, 2013·Expert Review of Pharmacoeconomics & Outcomes Research·Sheri FehnelAri Gnanasakthy
Feb 19, 2016·Nature Reviews. Cardiology·Daniel B Mark
Jun 28, 2014·Value in Health : the Journal of the International Society for Pharmacoeconomics and Outcomes Research·Tara SymondsLucy Abraham
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Apr 22, 2021·Journal of Patient-reported Outcomes·Kyungwan HongEleanor M Perfetto

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