The Use of Patient-Reported Outcome Measures in Rare Diseases and Implications for Health Technology Assessment

Background Patient-reported outcome measures (PROMs) are used in health technology assessment (HTA) to measure patient experiences with disease and treatment, allowing a deeper understanding of treatment impact beyond clinical endpoints. Developing and administering PROMs for rare diseases poses uni...

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Bibliographic Details
Published in:	The patient : patient-centered outcomes research Vol. 14; no. 5; pp. 485 - 503
Main Authors:	Whittal, Amanda, Meregaglia, Michela, Nicod, Elena
Format:	Journal Article
Language:	English
Published:	Cham Springer International Publishing 01.09.2021 Springer Nature B.V
Subjects:	Data collection Disease Health Administration Health Economics Health technology assessment Humans Medicine Medicine & Public Health Patient Reported Outcome Measures Patients Pharmacoeconomics and Health Outcomes Preferences Psychometrics Public Health Quality of Life Quality of Life Research Quantitative psychology Questionnaires Rare diseases Rare Diseases - therapy Review Review Article Technology Assessment, Biomedical
ISSN:	1178-1653, 1178-1661, 1178-1661
Online Access:	Get full text
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