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Lookup NU author(s): Stephen RiceORCiD, Dr Viviana AlbaniORCiD, Dr Dimitrios Minos, Dr Gulnar Fattakhova, Dr Mark ShirleyORCiD, Dr Diarmuid CoughlanORCiD, Professor Luke ValeORCiD, Professor David Jones
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Copyright © 2020 AGA Institute. Published by Elsevier Inc. All rights reserved. BACKGROUND & AIMS: There have been few high-quality studies of the costs, preference-based health-related quality of life (HRQoL) and cost effectiveness of treatments for primary biliary cholangitis (PBC). We aimed to estimate the marginal effects of PBC complications and symptoms, accounting for treatment, on HRQoL and the annual cost of health care in the United Kingdom (UK). These are essential components for evaluation of cost effectiveness and this information will aid in evaluation of new treatments. METHODS: Questionnaires were mailed to 4583 participants in the UK-PBC research cohort and data were collected on HRQoL and use of the National Health Service (NHS) in the UK from 2015 through 2016. HRQoL was measured using the EQ-5D-5L instrument. The annual cost of resource use was calculated using unit costs obtained from NHS sources. We performed econometric analyses to determine the effects of treatment, symptoms, complications, liver transplantation status, and patient characteristics on HRQoL and annual costs. RESULTS: In an analysis of data from 2240 participants (over 10% of all UK PBC patients), we found that PBC symptoms have a considerable effect on HRQoL. Ursodeoxycholic acid therapy was associated with significantly higher HRQoL regardless of response status. Having had a liver transplant and ascites were also independently associated with reduced HRQoL. Having had a liver transplant (US$4294) and esophageal varices (US$3401) were the factors with the two greatest mean annual cost to the NHS. Symptoms were not independently associated with cost but were associated with reduction in HRQoL for patients, indicating the lack of effective treatments for PBC symptoms. CONCLUSIONS: In an analysis of data from 2240 participants in the UK PBC, we found that HRQoL and cost estimates provide greater insight into the relative importance of PBC-related symptoms and complications. These findings provide estimates for health technology assessments of new treatments for PBC.
Author(s): Rice S, Albani V, Minos D, Fattakhova G, Mells GF, Carbone M, Flack S, Varvaropoulou N, Badrock J, Spicer A, Sandford RN, Shirley MDF, Coughlan D, Hirschfield G, Taylor-Robinson SD, Vale L, Jones DEJ
Publication type: Article
Publication status: Published
Journal: Clinical Gastroenterology and Hepatology
Year: 2021
Volume: 19
Issue: 4
Pages: 768-776.e10
Print publication date: 01/04/2021
Online publication date: 17/06/2020
Acceptance date: 11/06/2020
ISSN (print): 1542-3565
ISSN (electronic): 1542-7714
Publisher: Elsevier
URL: https://doi.org/10.1016/j.cgh.2020.06.025
DOI: 10.1016/j.cgh.2020.06.025
PubMed id: 32562892
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