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Abstract Details
Effects of Primary Biliary Cholangitis on Quality of Life and Health Care Costs in the United Kingdom
Clin Gastroenterol Hepatol. 2020 Jun 17;S1542-3565(20)30836-3.doi: 10.1016/j.cgh.2020.06.025. Online ahead of print.
Stephen Rice1, Viviana Albani2, Dimitrios Minos3, Gulnar Fattakhova2, George F Mells4, Marco Carbone4, Steven Flack4, Nikoletta Varvaropoulou4, Jonathan Badrock4, Ann Spicer4, Richard N Sandford4, Mark D F Shirley5, Diarmuid Coughlan2, Gideon Hirschfield6, Simon D Taylor-Robinson7, UK-PBC Consortium; Luke Vale2, David E J Jones8
Author information
1Health Economics Group, Institute of Health and Society, Newcastle University, UK. Electronic address: stephen.rice@ncl.ac.uk.
2Health Economics Group, Institute of Health and Society, Newcastle University, UK.
3Department of Political Economy, King's College London, UK.
4Department of Medical Genetics, Cambridge Biomedical Campus, University of Cambridge, UK.
5School of Biology, Newcastle University, UK.
6Institute of Immunology and Immunotherapy, University of Birmingham, UK; Toronto Centre for Liver Disease, University Health Network, University of Toronto, Toronto, Canada.
7Department of Surgery & Cancer, Imperial College London, UK.
8Institute of Cellular Medicine, Newcastle University, UK.
Abstract
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.