Predicted Lifetime Health Care Resource Use Costs Associated With The Treatment Of Patients With Primary Biliary Cholangitis From A UK Payer PerspectivE
Author(s)
Oyinkan Solanke, MSc1, James Gould, PhD2, Vicki Laskier-Owens, PhD2, Emily Combe, MSc2, Rikke Brandt, BSc, MSc1, Darren Asquith, PhD1.
1Ipsen, London, United Kingdom, 2Fiecon, London, United Kingdom.
1Ipsen, London, United Kingdom, 2Fiecon, London, United Kingdom.
OBJECTIVES: Primary biliary cholangitis (PBC), a rare, autoimmune, chronic, cholestatic liver disease, is associated with substantial direct health care resource use (HCRU) costs. Here we present HCRU costs associated with second-line PBC treatments.
METHODS: A 10-health state Markov cohort model was developed to model the progression of PBC over a lifetime horizon and estimate the incremental cost-effectiveness of elafibranor compared to other second-line treatment options for PBC, using an annual 3.5% discount rate. The model consisted of a PBC biomarker component, with states defined by alkaline phosphatase and total bilirubin levels, and a liver disease component which included the following states: decompensated cirrhosis, hepatocellular carcinoma, pre-liver transplant (LT), LT, post-LT and PBC re-emergence. The PBC biomarker component stratified patients by risk of progression to the liver disease component. The liver disease component contained patients who progressed to the states included herein. The ELATIVE clinical trial informed transition probabilities for elafibranor and ursodeoxycholic acid (UDCA). For obeticholic acid (OCA), transition probabilities were derived from indirect treatment comparisons with elafibranor. Literature informed transition probabilities between the liver disease states. HCRU and costs, inflated to 2023, were sourced from previous NICE appraisals, national databases and literature.
RESULTS: Elafibranor was associated with the lowest predicted lifetime HCRU costs compared to OCA and UDCA monotherapy (£64,004, £88,287 and £108,552, respectively) for second-line PBC treatment. Elafibranor accrued the lowest HCRU costs compared to OCA and UDCA in each component of the model: £21,838 (PBC biomarker) and £39,175 (liver disease), compared to OCA (£27,596 and £56,578) and UDCA (£32,585 and £71,287).
CONCLUSIONS: Elafibranor is associated with the lowest predicted lifetime HCRU costs amongst second-line treatment options for patients with PBC. This is due to a slower rate of progression to more severe PBC biomarker health states and liver disease health states for those treated with elafibranor.
METHODS: A 10-health state Markov cohort model was developed to model the progression of PBC over a lifetime horizon and estimate the incremental cost-effectiveness of elafibranor compared to other second-line treatment options for PBC, using an annual 3.5% discount rate. The model consisted of a PBC biomarker component, with states defined by alkaline phosphatase and total bilirubin levels, and a liver disease component which included the following states: decompensated cirrhosis, hepatocellular carcinoma, pre-liver transplant (LT), LT, post-LT and PBC re-emergence. The PBC biomarker component stratified patients by risk of progression to the liver disease component. The liver disease component contained patients who progressed to the states included herein. The ELATIVE clinical trial informed transition probabilities for elafibranor and ursodeoxycholic acid (UDCA). For obeticholic acid (OCA), transition probabilities were derived from indirect treatment comparisons with elafibranor. Literature informed transition probabilities between the liver disease states. HCRU and costs, inflated to 2023, were sourced from previous NICE appraisals, national databases and literature.
RESULTS: Elafibranor was associated with the lowest predicted lifetime HCRU costs compared to OCA and UDCA monotherapy (£64,004, £88,287 and £108,552, respectively) for second-line PBC treatment. Elafibranor accrued the lowest HCRU costs compared to OCA and UDCA in each component of the model: £21,838 (PBC biomarker) and £39,175 (liver disease), compared to OCA (£27,596 and £56,578) and UDCA (£32,585 and £71,287).
CONCLUSIONS: Elafibranor is associated with the lowest predicted lifetime HCRU costs amongst second-line treatment options for patients with PBC. This is due to a slower rate of progression to more severe PBC biomarker health states and liver disease health states for those treated with elafibranor.
Conference/Value in Health Info
2025-05, ISPOR 2025, Montréal, Quebec, CA
Value in Health, Volume 28, Issue S1
Code
EE187
Topic
Economic Evaluation
Topic Subcategory
Cost/Cost of Illness/Resource Use Studies
Disease
SDC: Rare & Orphan Diseases