Health Utility Values in Adults With Primary Biliary Cholangitis: A Global Systematic Literature Review
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, M.Sc.3, Sumeet Attri, M.Pharm4, Sukriti Sharma, M.Sc.4, 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: Primary Biliary Cholangitis (PBC), a rare progressive autoimmune liver disease that significantly impairs quality-of-life. Health Utility Values (HUVs), a critical parameter for economic evaluations (EE) and health technology assessments (HTA), remain limited and heterogeneous in PBC. This systematic literature review (SLR) aimed to identify and summarise the published global evidence on HUVs in adult patients with PBC.
METHODS: Embase® and MEDLINE®, mostly recommended by HTA agencies, were searched from inception to August 2024. Citations were screened in parallel by a human and a GPT-4-based AI, with any discrepancies resolved by a human subject matter expert.
RESULTS: Out of 292 records screened, seven studies (14 publications) were included, conducted in the UK (n=2), globally (n=2), the US (n=1), Italy (n=1), and Denmark (n=1). Study designs included prospective cohort (n=3), cross-sectional (n=3), and randomised controlled trial (n=1). EQ-5D index scale was mostly used (n=6), with one study using both HUI Mark-2 and 3. Overall, reported HUVs ranged from 0.28 to 0.87. Compared to the general population (HUVs: 0.82-0.95), PBC patients had consistently lower values (0.32-0.87). Higher HUVs were associated with standard of care (SoC) (incremental utility: 0.03, 95% CI: 0 to 0.05 vs. no SoC) and liver transplant (0.82 at 24 months post-transplant vs. 0.38 at listing for transplant). Conversely, lower HUVs were observed in PBC patients with depression (0.40), pruritus (0.49), and severe sleep disturbance (0.52), with further reductions in those experiencing both depression and severe pruritus (0.28-0.30).
CONCLUSIONS: HUVs in PBC populations are consistently lower than in the general population, particularly among individuals burdened by symptoms such as depression, pruritus, and sleep disturbances. These findings underscore the need to capture symptom-specific and treatment-responsive utility values to support robust health economic evaluations in PBC.
METHODS: Embase® and MEDLINE®, mostly recommended by HTA agencies, were searched from inception to August 2024. Citations were screened in parallel by a human and a GPT-4-based AI, with any discrepancies resolved by a human subject matter expert.
RESULTS: Out of 292 records screened, seven studies (14 publications) were included, conducted in the UK (n=2), globally (n=2), the US (n=1), Italy (n=1), and Denmark (n=1). Study designs included prospective cohort (n=3), cross-sectional (n=3), and randomised controlled trial (n=1). EQ-5D index scale was mostly used (n=6), with one study using both HUI Mark-2 and 3. Overall, reported HUVs ranged from 0.28 to 0.87. Compared to the general population (HUVs: 0.82-0.95), PBC patients had consistently lower values (0.32-0.87). Higher HUVs were associated with standard of care (SoC) (incremental utility: 0.03, 95% CI: 0 to 0.05 vs. no SoC) and liver transplant (0.82 at 24 months post-transplant vs. 0.38 at listing for transplant). Conversely, lower HUVs were observed in PBC patients with depression (0.40), pruritus (0.49), and severe sleep disturbance (0.52), with further reductions in those experiencing both depression and severe pruritus (0.28-0.30).
CONCLUSIONS: HUVs in PBC populations are consistently lower than in the general population, particularly among individuals burdened by symptoms such as depression, pruritus, and sleep disturbances. These findings underscore the need to capture symptom-specific and treatment-responsive utility values to support robust health economic evaluations in PBC.
Conference/Value in Health Info
2025-11, ISPOR Europe 2025, Glasgow, Scotland
Value in Health, Volume 28, Issue S2
Code
PCR110
Topic
Economic Evaluation, Health Technology Assessment, Patient-Centered Research
Topic Subcategory
Health State Utilities
Disease
Rare & Orphan Diseases, Systemic Disorders/Conditions (Anesthesia, Auto-Immune Disorders (n.e.c.), Hematological Disorders (non-oncologic), Pain)