ECONOMIC IMPACT OF EARLY DIAGNOSIS AND TREATMENT FOR INBORN ERRORS OF IMMUNITY WITH A FOCUS ON COMMON VARIABLE IMMUNODEFICIENCY (CVID) IN SPAIN: A COST-EFFECTIVENESS PERSPECTIVE
Author(s)
Batyrkhan Kuatov, MSc1, Annaëlle Vinzent, MSc2, Alessandro Catanzaro, MSc3, Melike INKAYA TASKIN, BSc, MBA4, Jonathan Galduf, MSc, PharmD5, Chrissy Lowry, MSc2, Pere Soler-Palacín, MD, PhD, MSc.6;
1CSL Behring, Bern, Switzerland, 2Lumanity, London, United Kingdom, 3Lumanity, Utrecht, Netherlands, 4CSL Behring, ISTANBUL, Turkey, 5CSL BEHRING, S.A., Barcelona, Spain, 6Hospital Universitari Vall d'Hebron, Barcelona, Catalonia, Spain
1CSL Behring, Bern, Switzerland, 2Lumanity, London, United Kingdom, 3Lumanity, Utrecht, Netherlands, 4CSL Behring, ISTANBUL, Turkey, 5CSL BEHRING, S.A., Barcelona, Spain, 6Hospital Universitari Vall d'Hebron, Barcelona, Catalonia, Spain
OBJECTIVES: Inborn errors of immunity (IEI) are genetic disorders impairing immune function, leading to severe/recurrent infections, inflammation, autoimmunity and cancer. Common variable immunodeficiency (CVID), a heterogeneous predominantly antibody deficiency, is the commonest symptomatic IEI. We assessed the cost-effectiveness and quality-of-life impact of early diagnosis and immunoglobulin replacement therapy (IGRT) versus delayed diagnosis for CVID in Spain.
METHODS: A cohort Markov model was developed from a Spanish payer perspective, with a societal perspective explored as a scenario. Patients transitioned across 13 health states defined by diagnosis and complication status. Health state-specific transition probabilities, mortality rates, costs and health utilities were derived from the literature and validated by a clinical expert. IGRT was modeled as standard treatment for diagnosed patients in a complication health state. The model assumed a 4-year median diagnosis delay, starting at age 1 year (20% vs. 70% diagnosed in early diagnosis cohort), a lifetime horizon with 28-day cycles, and 3% annual discount rate to costs and outcomes. Spain’s unofficial willingness-to-pay threshold (WTP) was assumed as €30,000/quality-adjusted life years (QALYs).
RESULTS: Early diagnosis added €79,123 in costs but yielded 3.021 additional life years and 2.997 additional QALYs, with an incremental cost-effectiveness ratio (ICER) of €26,402/QALY, below the Spanish WTP. The incremental cost was primarily driven by IGRT costs. However, these costs are partially compensated from savings in societal costs in the societal perspective scenario (~30% reduction in the early diagnosis arm), highlighting the broader implications of earlier diagnosis and IGRT beyond direct healthcare costs. Sensitivity analyses confirmed the robustness of base case findings, with early diagnosis being cost-effective in 71% of probabilistic iterations and key deterministic sensitivity analyses at the considered WTP.
CONCLUSIONS: Early diagnosis and timely IGRT for IEI was cost-effective in Spain from both payer and societal perspectives and the results were robust to the sensitivity analyses.
METHODS: A cohort Markov model was developed from a Spanish payer perspective, with a societal perspective explored as a scenario. Patients transitioned across 13 health states defined by diagnosis and complication status. Health state-specific transition probabilities, mortality rates, costs and health utilities were derived from the literature and validated by a clinical expert. IGRT was modeled as standard treatment for diagnosed patients in a complication health state. The model assumed a 4-year median diagnosis delay, starting at age 1 year (20% vs. 70% diagnosed in early diagnosis cohort), a lifetime horizon with 28-day cycles, and 3% annual discount rate to costs and outcomes. Spain’s unofficial willingness-to-pay threshold (WTP) was assumed as €30,000/quality-adjusted life years (QALYs).
RESULTS: Early diagnosis added €79,123 in costs but yielded 3.021 additional life years and 2.997 additional QALYs, with an incremental cost-effectiveness ratio (ICER) of €26,402/QALY, below the Spanish WTP. The incremental cost was primarily driven by IGRT costs. However, these costs are partially compensated from savings in societal costs in the societal perspective scenario (~30% reduction in the early diagnosis arm), highlighting the broader implications of earlier diagnosis and IGRT beyond direct healthcare costs. Sensitivity analyses confirmed the robustness of base case findings, with early diagnosis being cost-effective in 71% of probabilistic iterations and key deterministic sensitivity analyses at the considered WTP.
CONCLUSIONS: Early diagnosis and timely IGRT for IEI was cost-effective in Spain from both payer and societal perspectives and the results were robust to the sensitivity analyses.
Conference/Value in Health Info
2026-05, ISPOR 2026, Philadelphia, PA, USA
Value in Health, Volume 29, Issue S6
Code
EE276
Topic
Economic Evaluation
Disease
SDC: Rare & Orphan Diseases