ECONOMIC IMPACT OF EARLY DIAGNOSIS AND IMMUNOGLOBULIN TREATMENT FOR INBORN ERRORS OF IMMUNITY WITH FOCUS ON COMMON VARIABLE IMMUNODEFICIENCY (CVID) IN BRAZIL: A COST-EFFECTIVENESS PERSPECTIVE
Author(s)
Batyrkhan Kuatov, MA1, Annaëlle Vinzent, MSc2, Autumn Hoang, MSc2, Melike Inkaya Taskin, Sr., EMAUD3, Chrissy Lowry, MSc2, Lilian Pititto, MBA4, Ekaterini Simões Goudouris, PhD, MD5.
1Director HEOR, Global Market Access, CSL Behring, Bern, Switzerland, 2Lumanity, London, United Kingdom, 3CSL BEHRING, Istanbul, Turkey, 4CSL Behring, Sao Paulo, Brazil, 5FEDERAL UNIVERSITY OF RIO DE JANEIRO, Rio de Janeiro, Brazil.
1Director HEOR, Global Market Access, CSL Behring, Bern, Switzerland, 2Lumanity, London, United Kingdom, 3CSL BEHRING, Istanbul, Turkey, 4CSL Behring, Sao Paulo, Brazil, 5FEDERAL UNIVERSITY OF RIO DE JANEIRO, Rio de Janeiro, Brazil.
OBJECTIVES: Inborn errors of immunity (IEI), also known as primary immunodeficiencies, are genetic disorders of immune function associated with recurrent or severe infections, inflammation, autoimmunity and malignancy. Common variable immunodeficiency (CVID), a rare heterogeneous antibody deficiency, is the most common symptomatic IEI. We assessed the cost-effectiveness and health-related quality-of-life impact of early diagnosis and immunoglobulin replacement therapy (IGRT) versus delayed diagnosis for CVID in Brazil.
METHODS: The model was adapted from a published Spanish cohort Markov model and presents a public payer perspective in the base case. Within the model, 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. The model used a lifetime time horizon with 28-day cycles, applying a 5% annual discount rate to costs and outcomes. Cost-effectiveness was assessed against Brazil’s willingness-to-pay (WTP) threshold of R$120,000/quality-adjusted life years (QALYs). IGRT was modelled as standard treatment for diagnosed patients in a complication health state.
RESULTS: Early diagnosis added R$103,735 in costs but yielded 2.31 additional life years and 2.40 additional QALYs compared with delayed diagnosis, with an incremental cost-effectiveness ratio (ICER) of R$43,259/QALY gained, well below the Brazilian WTP threshold for rare diseases. The incremental cost was primarily driven by IGRT costs, and incremental QALYs were driven by increased time spent in asymptomatic states in the early diagnosis arm. Scenario analyses showed that increasing subcutaneous IGRT use to 30% reduced the ICER to R$32,386, suggesting that broader adoption of subcutaneous IGRT therapy could further improve the cost-effectiveness.
CONCLUSIONS: Early diagnosis and timely IGRT for IEI were cost-effective in Brazil, with ICERs well below the R$120,000/QALY threshold.
METHODS: The model was adapted from a published Spanish cohort Markov model and presents a public payer perspective in the base case. Within the model, 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. The model used a lifetime time horizon with 28-day cycles, applying a 5% annual discount rate to costs and outcomes. Cost-effectiveness was assessed against Brazil’s willingness-to-pay (WTP) threshold of R$120,000/quality-adjusted life years (QALYs). IGRT was modelled as standard treatment for diagnosed patients in a complication health state.
RESULTS: Early diagnosis added R$103,735 in costs but yielded 2.31 additional life years and 2.40 additional QALYs compared with delayed diagnosis, with an incremental cost-effectiveness ratio (ICER) of R$43,259/QALY gained, well below the Brazilian WTP threshold for rare diseases. The incremental cost was primarily driven by IGRT costs, and incremental QALYs were driven by increased time spent in asymptomatic states in the early diagnosis arm. Scenario analyses showed that increasing subcutaneous IGRT use to 30% reduced the ICER to R$32,386, suggesting that broader adoption of subcutaneous IGRT therapy could further improve the cost-effectiveness.
CONCLUSIONS: Early diagnosis and timely IGRT for IEI were cost-effective in Brazil, with ICERs well below the R$120,000/QALY threshold.
Conference/Value in Health Info
2026-11, ISPOR Europe 2026, Vienna, Austria
Value in Health, Volume 29, Issue 12S
Code
EE561
Topic
Economic Evaluation
Disease
Rare & Orphan Diseases