Patient-reported outcomes to support medical product labeling claims: FDA perspective.
By D.walaa badawy, L. Burke, J. Powers, Jane A. Scott, E. Rock, Sahar Dawisha, R. O'neill, D. Kennedy. Value in Health, 2007
699 citations recorded by the source corpus.
This article concerns development and use of patient-reported outcomes (PROs) in clinical trials to evaluate medical products. A PRO is any report coming directly from patients, without interpretation by physicians or others, about how they function or feel in relation to a health condition and its therapy. PRO instruments are used to measure these patient reports. PROs provide a unique perspective on medical therapy, because some effects of a health condition and its therapy are known only to patients. Properly developed and evaluated PRO instruments also have the potential to provide more sensitive and specific measurements of the effects of medical therapies, thereby increasing the efficiency of clinical trials that attempt to measure the meaningful treatment benefits of those therapies. Poorly developed and evaluated instruments may provide misleading conclusions or data that cannot be used to support product labeling claims. We review selected major challenges from Food and Drug Administration's perspective in using PRO instruments, measures, and end points to support treatment benefit claims in product labeling. These challenges highlight the need for sponsors to formulate desired labeling claim(s) prospectively, to acquire and document information needed to support these claim(s), and to identify existing instruments or develop new and more appropriate PRO instruments for evaluating treatment benefit in the defined population in which they will seek claims.
Subfields: Economics and Econometrics, Statistics and Probability
View Paper: https://www.semanticscholar.org/paper/e4a5111030d72cd3e13ae331125d212df31389c5
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Citations in the corpus
- Multinational trials-recommendations on the translations required, approaches to using the same language in different countries, and the approaches to support pooling the data: the ISPOR Patient-Reported Outcomes Translation and Linguistic Validation Good Research Practices Task Force report. (2009, background): “Patrick et al. [ 26 ] have suggested that in order to verify basic measurement properties, such as distribution of responses, internal consistency, test‐retest reliability, and validity across multiple languages, it is necessary to consider population characteristics and variability around the data,”
- Qualitative research and content validity: developing best practices based on science and experience (2009, background): “In FDA terminology, each concept corresponds to a medical product labeling claim, which the PRO may be used to support [25].”