C-PEPTIDE-BASED ECONOMIC MODEL FOR EVALUATING TREGS PLUS RITUXIMAB IN RECENT-ONSET TYPE 1 DIABETES
Author(s)
Diana Teloian, MA1, Marcin Kądziela, MSc2, Marta Druch, MSc, MBA2, Karol Ptach, MSc, MBA2, Katarzyna Karpińska, MSc2, Ewelina Bróskowska, MSc2, Mariusz Jabłoński, MD2, Piotr Trzonkowski, MD, PhD2.
1Parexel International, Tbilisi, Georgia, 2PolTREG S.A., Gdańsk, Poland.
1Parexel International, Tbilisi, Georgia, 2PolTREG S.A., Gdańsk, Poland.
OBJECTIVES: Type 1 diabetes (T1D) is characterized by progressive loss of beta-cell function (BCF), leading to lifelong insulin dependence and substantial burden. C-peptide is a widely used marker of BCF and surrogate endpoint in disease-modifying trials for recent-onset stage 3 T1D, where greater preservation is associated with improved metabolic outcomes. Recent literature identifies C-peptide as an important prognostic biomarker not yet adequately addressed in pediatric economic evaluations. This study develops a novel C-peptide-based health state cost-effectiveness model for early-onset T1D.
METHODS: A Markov cohort model was developed with a lifetime horizon and annual cycle length. The model was designed to enable economic comparison of the disease-modifying treatment Tregs plus rituximab added to insulin therapy versus standard of care (insulin therapy). The model defined two living health states based on stimulated C-peptide thresholds (Preserved BCF [≥0.2 nmol/L] and Loss of BCF [<0.2 nmol/L]) and death state. The cohort entered at age 12.8 years with recent-onset T1D and preserved BCF (0.86 nmol/L). Transitions to Loss of BCF state were driven by biphasic C-peptide decline using piecewise exponential decay: steep Phase 1 decline (0-6.9 years) estimated from a log-linear fit to TregVAC2.0 clinical trial data, followed by slower Phase 2 decline (6.9+ years) based on published observational data. Mortality and utility were based on published literature. Costs and outcomes were aggregated per health state. Outcomes were discounted at 3.5%. The results are illustrated using the standard-of-care reference case.
RESULTS: For standard of care, the model projected 23.47 discounted life years (2.35 in Preserved BCF state) and 20.77 quality-adjusted life years. Deterministic sensitivity analyses identified C-peptide decline rates and mortality as primary outcome drivers.
CONCLUSIONS: This novel C-peptide-based model provides a transparent, robust framework for evaluating the cost-effectiveness of disease-modifying treatments in pediatric early-onset T1D by directly linking BCF preservation to health outcomes.
METHODS: A Markov cohort model was developed with a lifetime horizon and annual cycle length. The model was designed to enable economic comparison of the disease-modifying treatment Tregs plus rituximab added to insulin therapy versus standard of care (insulin therapy). The model defined two living health states based on stimulated C-peptide thresholds (Preserved BCF [≥0.2 nmol/L] and Loss of BCF [<0.2 nmol/L]) and death state. The cohort entered at age 12.8 years with recent-onset T1D and preserved BCF (0.86 nmol/L). Transitions to Loss of BCF state were driven by biphasic C-peptide decline using piecewise exponential decay: steep Phase 1 decline (0-6.9 years) estimated from a log-linear fit to TregVAC2.0 clinical trial data, followed by slower Phase 2 decline (6.9+ years) based on published observational data. Mortality and utility were based on published literature. Costs and outcomes were aggregated per health state. Outcomes were discounted at 3.5%. The results are illustrated using the standard-of-care reference case.
RESULTS: For standard of care, the model projected 23.47 discounted life years (2.35 in Preserved BCF state) and 20.77 quality-adjusted life years. Deterministic sensitivity analyses identified C-peptide decline rates and mortality as primary outcome drivers.
CONCLUSIONS: This novel C-peptide-based model provides a transparent, robust framework for evaluating the cost-effectiveness of disease-modifying treatments in pediatric early-onset T1D by directly linking BCF preservation to health outcomes.
Conference/Value in Health Info
2026-11, ISPOR Europe 2026, Vienna, Austria
Value in Health, Volume 29, Issue 12S
Code
EE170
Topic
Economic Evaluation, Health Technology Assessment
Topic Subcategory
Trial-Based Economic Evaluation
Disease
Diabetes/Endocrine/Metabolic Disorders (including obesity)