Journal article
Health-related quality of life and a cost-utility simulation of adults in the UK with osteogenesis imperfecta, X-linked hypophosphatemia and fibrous dysplasia
L Forestier-Zhang, L Watts, A Turner, H Teare, J Kaye, J Barrett, C Cooper, R Eastell, P Wordsworth, MK Javaid, R Pinedo-Villanueva
Orphanet Journal of Rare Diseases | BMC | Published : 2016
Abstract
Background: Health-related quality of life of adults with osteogenesis imperfecta (OI), fibrous dysplasia (FD) and X-linked hypophosphatemia (XLH) remains poorly described. The aim of this study was to describe the HRQoL of adults with osteogenesis imperfecta, fibrous dysplasia and X-linked hypophophataemia and perform a cost-utility simulation to calculate the maximum cost that a health care system would be willing to pay for a hypothetical treatment of a rare bone disease. Results: Participants completed the EQ-5D-5 L questionnaire between September 2014 and March 2016. For the economic simulation, we considered a hypothetical treatment that would be applied to OI participants in the lower..
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Awarded by Medical Research Council
Funding Acknowledgements
The RUDY study was funded by the UK National Institute of Health Research (NIHR) Rare Diseases Translational Research Collaboration in Musculoskeletal Diseases and the Oxford NIHR Musculoskeletal Biomedical Research Unit. The funders had no role in the design of the study and collection, analysis, and interpretation of data and in writing the manuscript.