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 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.
| Original language | English |
|---|---|
| Pages (from-to) | 768-776.e10 |
| Number of pages | 9 |
| Journal | Clinical Gastroenterology and Hepatology |
| Volume | 19 |
| Issue number | 4 |
| Early online date | 17 Jun 2020 |
| DOIs | |
| Publication status | Published - 1 Apr 2021 |
Bibliographical note
Funding Information:Conflicts of interest These authors disclose the following: Prof Jones reports grants, personal fees, and other from Intercept, other from GSK, other from Novartis, other from Cymabay, other from FF Pharma, other from Shire, and grants and other from Pfizer outside the submitted work. Dr Mells reports grants from Medical Research Council, UK, grants from National Institute for Health Research Rare Diseases - Translational Research Collaboration, during the conduct of the study, and personal fees from Intercept Pharmaceuticals outside the submitted work. Dr Hirschfield reports personal fees from Intercept Pharma, personal fees from Cymabay, grants and personal fees from Falk Pharma, personal fees from GSK, personal fees from Novartis, and grants from Gilead outside the submitted work. Prof Luke Vale reports grants from Medical Research Council, UK. The remaining authors disclose no conflicts.
Funding Information:
Funding Supported by the UK Medical Research Council (MRC).
Publisher Copyright:
© 2021 AGA Institute
Keywords
- Cost of Illness
- Economics
- Management
- UDCA