The US Food and Drug Administration published a draft guidance document in 2006, which was finalized in 2009, that established the regulatory standards for development, validation, and use of patient-reported outcome (PRO) measures to support medical product–labeling claims regarding treatment benefit (ie, efficacy or comparative safety).1 The principles outlined in this document represent the best practices in the field; however, the requirement to provide evidence to support validity and reliability of PROs in the regulatory context represented a substantial change. This document has increased the scientific rigor of patient reports, which are being used as a primary basis for regulatory approval of medical products in some therapeutic areas (eg, some gastrointestinal diseases). In other therapeutic areas, such as oncology, there has been an increasing trend to consider the patient perspective in evaluation of medical products (eg, symptoms of myelofibrosis,2 pain in prostate cancer3). The framework outlined in the US Food and Drug Administration guidance document can also be applied more broadly to other types of clinical outcome assessments, including observer-based assessments and physician rating scales. Requiring evidence to support validity and reliability of physician rating scales in the regulatory context may well be the next frontier to be tackled across therapeutic areas.
Miskala PH. Development, Use, and Interpretation of Patient-Reported Outcomes for Clinical Decision Making in Ophthalmology. JAMA Ophthalmol. 2016;134(6):690–692. doi:10.1001/jamaophthalmol.2016.0820
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