Immunoglobulin Replacement Therapy is critical and cost-effective in increasing life expectancy and quality of life in patients suffering from Common Variable Immunodeficiency Disorders (CVID): A health-economic assessment

• Published in: PloS one Vol. 16; no. 3; p. e0247941
• Main Authors: van Wilder, Philippe, Odnoletkova, Irina, Mouline, Mehdi, de Vries, Esther
• Format: Journal Article
• Language: English
• Published: United States Public Library of Science 04-03-2021; Public Library of Science (PLoS)
• Subjects: Antibodies; Biology and Life Sciences; Care and treatment; Chronic illnesses; Clinical decision making; Common variable immunodeficiency; Common Variable Immunodeficiency - economics; Common Variable Immunodeficiency - therapy; Cost analysis; Cost-Benefit Analysis; Decision making; Disease; Economic analysis; Economic aspects; Economic models; Economics; Editing; Estimates; Genetic aspects; Health economics; Humans; Immunoglobulin G; Immunoglobulins; Immunoglobulins - administration & dosage; Immunoglobulins - economics; Immunoglobulins - therapeutic use; Immunological deficiency syndromes; Immunology; Infections; Life Expectancy; Life span; Mean; Medicine and Health Sciences; Meta-analysis; Methodology; Microbiology; Models, Economic; Morbidity; Mortality; Patients; Physical sciences; Physiological aspects; Pneumonia; Public health; Quality of Life; Quality-Adjusted Life Years; Rare diseases; Regulatory agencies; Research and analysis methods; Research facilities; Social Sciences; Society; Testing; Treatment Outcome; Visualization; Belgium; Netherlands
• ISSN: 1932-6203; 1932-6203
• DOI: 10.1371/journal.pone.0247941

Common variable immunodeficiency disorders (CVID), the most common form of primary antibody deficiency, are rare conditions associated with considerable morbidity and mortality. The clinical benefit of immunoglobulin replacement therapy (IgGRT) is substantial: timely treatment with appropriate doses significantly reduces mortality and the incidence of CVID-complications such as major infections and bronchiectasis. Unfortunately, CVID-patients still face a median diagnostic delay of 4 years. Their disease burden, expressed in annual loss of disability-adjusted life years, is 3-fold higher than in the general population. Hurdles to treatment access and reimbursement by healthcare payers may exist because the value of IgGRT is poorly documented. This paper aims to demonstrate cost-effectiveness and cost-utility (on life expectancy and quality) of IgGRT in CVID.
With input from a literature search, we built a health-economic model for cost-effectiveness and cost-utility assessment of IgGRT in CVID. We compared a mean literature-based dose (≥450mg/kg/4wks) to a zero-or-low dose (0 to ≤100 mg/kg/4wks) in a simulated cohort of adult patients from time of diagnosis until death; we also estimated the economic impact of diagnostic delay in this simulated cohort. Compared to no or minimal treatment, IgGRT showed an incremental benefit of 17 life-years (LYs) and 11 quality-adjusted life-years (QALYs), resulting in an incremental cost-effectiveness ratio (ICER) of €29,296/LY and €46,717/QALY. These results were robust in a sensitivity analysis. Reducing diagnostic delay by 4 years provided an incremental benefit of six LYs and four QALYs compared to simulated patients with delayed IgGRT initiation, resulting in an ICER of €30,374/LY and €47,495/QALY.
The health-economic model suggests that early initiation of IgGRT compared to no or delayed IgGRT is highly cost-effective. CVID-patients' access to IgGRT should be facilitated, not only because of proven clinical efficacy, but also due to the now demonstrated cost-effectiveness.