viernes, 24 de febrero de 2017

Health-related quality of life and a cost-utility simulation of adults in the UK with osteogenesis imperfecta, X-linked hypophosphatemia and fibrous dysplasia | Orphanet Journal of Rare Diseases | Full Text

Health-related quality of life and a cost-utility simulation of adults in the UK with osteogenesis imperfecta, X-linked hypophosphatemia and fibrous dysplasia | Orphanet Journal of Rare Diseases | Full Text

Orphanet Journal of Rare Diseases - IMPACT FACTOR 3.29

Health-related quality of life and a cost-utility simulation of adults in the UK with osteogenesis imperfecta, X-linked hypophosphatemia and fibrous dysplasia

  • Lydia Forestier-Zhang,
  • Laura Watts,
  • Alison Turner,
  • Harriet Teare,
  • Jane Kaye,
  • Joe Barrett,
  • Cyrus Cooper,
  • Richard Eastell,
  • Paul Wordsworth,
  • Muhammad K. JavaidEmail author and
  • Rafael Pinedo-Villanueva
Orphanet Journal of Rare Diseases201611:160
DOI: 10.1186/s13023-016-0538-4
Received: 23 August 2016
Accepted: 15 November 2016
Published: 28 November 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 tertile of the health utility score.
A total of 109 study participants fully completed the EQ-5D-5 L questionnaire (response rate 63%). Pain/discomfort was the most problematic domain for participants with all three diseases (FD 31%, XLH 25%, OI 16%).
The economic simulation identified an expected treatment impact of +2.5 QALYs gained per person during the 10-year period, which led to a willing to pay of £14,355 annually for a health care system willing to pay up to £50,000 for each additional QALY gained by an intervention.

Conclusions

This is the first study to quantitatively measure and compare the HRQoL of adults with OI, FD and XLH and the first to use such data to conduct an economic simulation leading to healthcare system willingness-to-pay estimates for treatment of musculoskeletal rare diseases at various cost-effectiveness thresholds.

Keywords

Quality of life Economic evaluation Osteogenesis imperfecta Fibrous dysplasia McCune Albright syndrome X-linked hypophosphatemia


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