Effects of Primary Biliary Cholangitis on Quality of Life and Health Care Costs in the United Kingdom
Open accessAbstract
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 costs 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.
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More than 80% of UDCA-treated patients continue to experience symptoms, especially fatigue and cognitive symptoms.
“>80% continue to experience symptoms, especially fatigue and cognitive symptoms that are seemingly not modified by any current intervention.2 3 9 17”
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- Authors
- Rice, S., Albani, V., Minos, D., Fattakhova, G., Mells, G. F., Carbone, M., Flack, S., Varvaropoulou, N., Badrock, J., Spicer, A., Sandford, R. N., Shirley, M. D. F., Coughlan, D., Hirschfield, G., Taylor-Robinson, S. D., Vale, L., & Jones, D. E. J.
- Affiliations
- Newcastle University (GB)
- King's College London (GB)
- University of Cambridge (GB)
- University Health Network (CA)
- Imperial College London (GB)
- Corresponding author
- Rice, Stephen
- ORCID
- Venue
- Clinical Gastroenterology and Hepatology
- Publisher
- Elsevier BV
- Published
- 2021-04
- Volume
- 19
- Issue
- 4
- Pages
- 768-776.e10
- ISSN
- 1542-3565 (ISSN-L)
- OpenAlex
- W3036264843
- Cited by
- 32
- References
- 24
- Citations by year
- 2026: 12 · 2025: 8 · 2024: 2 · 2023: 2 · 2022: 6 · 2020: 2
- FWCI
- 1.49
- Type
- journal article
- Language
- en
- Open access
- green
- hdl.handle.net
- Licence
- Locations
- Clinical Gastroenterology and Hepatology (publishedVersion)
- PubMed (publishedVersion)
- BOA (University of Milano-Bicocca) (submittedVersion)
- Spiral (Imperial College London) (submittedVersion, cc-by-nc-nd)
- Newcastle University ePrints (Newcastle Univesity)
- Funders
- Medical Research Council
- Topics
- Health Sciences › Medicine › Hepatology › Liver Diseases and Immunity
- Social Sciences › Economics, Econometrics and Finance › Economics and Econometrics › Health Systems, Economic Evaluations, Quality of Life
- Health Sciences › Medicine › Surgery › Pediatric Hepatobiliary Diseases and Treatments
- Keywords
- primary biliary cirrhosis, health-related quality of life, health care costs, cost-effectiveness, ursodeoxycholic acid, liver transplantation, ascites, esophageal varices, EQ-5D-5L, National Health Service, health technology assessment
- MeSH terms
- United Kingdom, Humans, Liver Cirrhosis, Biliary, Quality of Life, State Medicine, Health Care Costs
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