Humanistic and Economic Burden of Primary Biliary Cholangitis: A Systematic Review of Health-Related Quality of Life Healthcare Resource Utilization and Associated Cost
Author(s)
Dilip Makhija, MS1, Marvin Rock, DrPH, MPH1, Chong H Kim, MPH, MS, PhD1, Caroline Burk, PhD1, Mirko von Hein, M.Sc.2, Ryan Thaliffdeen, PharmD, MS1, Oskar Eklund, MSc3, Sumeet Attri, M.Pharm4, Gagandeep Kaur, M.Pharm4, Barinder Singh, RPh4.
1Gilead Sciences, Inc., Foster City, CA, USA, 2Gilead Sciences, London, United Kingdom, 3Gilead Sciences AB, Solna, Sweden, 4Pharmacoevidence, Mohali, India.
1Gilead Sciences, Inc., Foster City, CA, USA, 2Gilead Sciences, London, United Kingdom, 3Gilead Sciences AB, Solna, Sweden, 4Pharmacoevidence, Mohali, India.
OBJECTIVES: This systematic literature review aimed to summarize published evidence regarding health-related quality of life (HRQoL), healthcare resource utilization (HCRU), and associated costs in adult patients with primary biliary cholangitis (PBC).
METHODS: Embase®, and MEDLINE®, and CENTRAL were searched from database inception to August 2024 using relevant keywords. Evidence was screened using both a manual approach and a Gen-AI tool, with quality check by a subject matter expert (human) as per NICE, UK and CDA, Canada position papers.
RESULTS: Out of 2,714 publications, 91 studies were included (HRQoL=68, HCRU & Cost=31 studies). Frequently used HRQoL instruments (≥5 studies) included PBC-40 (n=32), SF-36 (n=21), Fatigue Impact Scale (n=16), Pruritus Numerical Rating Scale (n=8), PBC-27 (n=5), and Chronic Liver Disease Questionnaire (n=5). HRQoL was significantly impaired in adult PBC patients compared to controls (healthy/non-PBC), with fatigue, social, emotional, and mental health as the most affected domains. Moderate to severe pruritus further worsened HRQoL compared to those with mild pruritus. PBC patients demonstrated increased HCRU, with longer hospital stays, more outpatient visits, and higher pharmacy use. Patients with alkaline phosphatase ≥1.5× ULN and cirrhosis were associated with significantly higher HCRU. Annual direct costs were significantly higher in patients with PBC compared to those without PBC. Key cost drivers included inpatient care, outpatient visits, liver transplantation (LT), and medications. Second-line treatment with obeticholic acid (OCA)/fibrates ± ursodeoxycholic acid (UDCA) incurred the highest total direct cost, followed by untreated and first-line (UDCA). Untreated patients incurred higher inpatient costs, largely due to liver transplantation, while in treated patients, medication expenses (OCA/fibrates, UDCA) were the main cost drivers.
CONCLUSIONS: PBC significantly impairs HRQoL and imposes a substantial economic burden due to high HCRU and treatment costs. This review underscores the critical need for timely and effective therapies that prioritize disease modification, symptom management, and overall patient well-being.
METHODS: Embase®, and MEDLINE®, and CENTRAL were searched from database inception to August 2024 using relevant keywords. Evidence was screened using both a manual approach and a Gen-AI tool, with quality check by a subject matter expert (human) as per NICE, UK and CDA, Canada position papers.
RESULTS: Out of 2,714 publications, 91 studies were included (HRQoL=68, HCRU & Cost=31 studies). Frequently used HRQoL instruments (≥5 studies) included PBC-40 (n=32), SF-36 (n=21), Fatigue Impact Scale (n=16), Pruritus Numerical Rating Scale (n=8), PBC-27 (n=5), and Chronic Liver Disease Questionnaire (n=5). HRQoL was significantly impaired in adult PBC patients compared to controls (healthy/non-PBC), with fatigue, social, emotional, and mental health as the most affected domains. Moderate to severe pruritus further worsened HRQoL compared to those with mild pruritus. PBC patients demonstrated increased HCRU, with longer hospital stays, more outpatient visits, and higher pharmacy use. Patients with alkaline phosphatase ≥1.5× ULN and cirrhosis were associated with significantly higher HCRU. Annual direct costs were significantly higher in patients with PBC compared to those without PBC. Key cost drivers included inpatient care, outpatient visits, liver transplantation (LT), and medications. Second-line treatment with obeticholic acid (OCA)/fibrates ± ursodeoxycholic acid (UDCA) incurred the highest total direct cost, followed by untreated and first-line (UDCA). Untreated patients incurred higher inpatient costs, largely due to liver transplantation, while in treated patients, medication expenses (OCA/fibrates, UDCA) were the main cost drivers.
CONCLUSIONS: PBC significantly impairs HRQoL and imposes a substantial economic burden due to high HCRU and treatment costs. This review underscores the critical need for timely and effective therapies that prioritize disease modification, symptom management, and overall patient well-being.
Conference/Value in Health Info
2025-11, ISPOR Europe 2025, Glasgow, Scotland
Value in Health, Volume 28, Issue S2
Code
EE524
Topic
Economic Evaluation, Patient-Centered Research, Real World Data & Information Systems
Topic Subcategory
Cost/Cost of Illness/Resource Use Studies
Disease
Rare & Orphan Diseases, Systemic Disorders/Conditions (Anesthesia, Auto-Immune Disorders (n.e.c.), Hematological Disorders (non-oncologic), Pain)