Value in health : the journal of the International Society for Pharmacoeconomics and Outcomes Research
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Comparative Study
The EUROHIS-QOL 8-item index: comparative psychometric properties to its parent WHOQOL-BREF.
To test the psychometric properties of the EUROHIS-QOL 8-item index, a shortened version of the World Health Organization Quality of Life Instrument-Abbreviated Version (WHOQOL-BREF). ⋯ The EUROHIS-QOL 8-item index showed acceptable cross-cultural performance and a satisfactory discriminant validity and would be a useful measure to include in studies to assess treatment effectiveness.
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In 2004, Willke and colleagues reviewed the efficacy endpoints reported in the labels of new drugs approved in the United States from 1997 through 2002 to evaluate the use of patient-reported outcome (PRO) endpoints. Of the labels reviewed, 30% included PROs. Our study aimed to build on this work by describing the current state of PRO label claims granted for new molecular entities (and biologic license applications since February 2006 after the release of the US Food and Drug Administration (FDA) draft PRO guidance. ⋯ The FDA continues to approve PRO claims, with 24% of new molecular entities and biologic license applications being granted. Successful PRO label claims over the past 5 years have generally supported treatment benefit for symptoms specified as primary endpoints.
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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. ⋯ Based on drug approval packages, nearly half (45%) of new molecular entitity/biologic license application products in the years 2006 to 2010 included PROs in the clinical trials supporting their approval, yet this rate is not reflected by claims granted. Understanding the nature of PRO claims granted under the current regulatory guidance is important. In addition, a clear understanding of denied claims yields valuable insight into where sponsors may improve implementation of PROs in clinical trials and submission of PRO evidence to increase the likelihood of obtaining PRO label claims.