ECONOMIC BURDEN OF ONE-YEAR MANAGEMENT OF PEDIATRIC COMBINED IMMUNODEFICIENCY: AN ACTIVITY-BASED COSTING ANALYSIS
Author(s)
Emna Dhaouedi, PharmD1, IKRAM FAZAA, PharmD2, Yosr Trabelsi, PharmD1, leila Achour, MSc2, Hend Chaker, PharmD3, Myriam Khrouf, MSc1, Hajer Felfel, III, MSc1, Monia Ouderni, MSc4, CHEMA DRIRA, MASc, MSc, PharmD2.
1university of pharmacy of monastir, Tunis, Tunisia, 2University of Tunis Manar, LR18ES39, Tunis, Tunisia, 3Genomics and oncogenetics research laboratory, Institute Pasteur of Tunis, Tunis, Tunisia, Tunis, United Arab Emirates, 4Bone Marrow Transplant Center of Tunisia, Tunis, Tunisia.
1university of pharmacy of monastir, Tunis, Tunisia, 2University of Tunis Manar, LR18ES39, Tunis, Tunisia, 3Genomics and oncogenetics research laboratory, Institute Pasteur of Tunis, Tunis, Tunisia, Tunis, United Arab Emirates, 4Bone Marrow Transplant Center of Tunisia, Tunis, Tunisia.
OBJECTIVES: Severe Combined immunodeficiency (SCID) is a severe form of primary immunodeficiency requiring intensive and sustained medical management. Evidence on its economic burden in low- and middle-income countries remains limited. This study aims to estimate costs related to one-year management of pediatric SCID (Severe combined immunodeficiency) patients from a hospital’s perspective and considered direct medical.
METHODS: This pharmaco-economic study was conducted at a Pediatric Hematology and Transplantation department, using an Activity-Based Costing (ABC) method. The one-year care pathway was mapped based on routine clinical practice and included outpatient visits, treatment (drugs and medical devices), biological and radiological monitoring, and healthcare resource utilization.
RESULTS: The total direct medical cost of SCID management was estimated at 23,914.12 € per patient. Drug therapy was the main cost driver (94.6%), amounting to 22 631.58€ , largely due to immunoglobulin replacement therapy. Biological and radiological monitoring accounted for 4.64% and was estimated at 1 109 ,86 €. Medical devices, nutrition, and personnel costs contributed marginally, representing 0.30 % , 0.20 %, and 0.23 % , respectively.
CONCLUSIONS: The management of pediatric SCID imposes a substantial economic burden in this setting, largely driven by high-cost immunoglobulin therapy. These findings highlight the need for cost-optimization strategies, including rational use and potential alternatives for immunoglobulin therapy. These results provide relevant evidence to inform reimbursement decisions and support sustainable access to essential treatments.
METHODS: This pharmaco-economic study was conducted at a Pediatric Hematology and Transplantation department, using an Activity-Based Costing (ABC) method. The one-year care pathway was mapped based on routine clinical practice and included outpatient visits, treatment (drugs and medical devices), biological and radiological monitoring, and healthcare resource utilization.
RESULTS: The total direct medical cost of SCID management was estimated at 23,914.12 € per patient. Drug therapy was the main cost driver (94.6%), amounting to 22 631.58€ , largely due to immunoglobulin replacement therapy. Biological and radiological monitoring accounted for 4.64% and was estimated at 1 109 ,86 €. Medical devices, nutrition, and personnel costs contributed marginally, representing 0.30 % , 0.20 %, and 0.23 % , respectively.
CONCLUSIONS: The management of pediatric SCID imposes a substantial economic burden in this setting, largely driven by high-cost immunoglobulin therapy. These findings highlight the need for cost-optimization strategies, including rational use and potential alternatives for immunoglobulin therapy. These results provide relevant evidence to inform reimbursement decisions and support sustainable access to essential treatments.
Conference/Value in Health Info
2026-11, ISPOR Europe 2026, Vienna, Austria
Value in Health, Volume 29, Issue 12S
Code
EE646
Topic
Economic Evaluation
Topic Subcategory
Cost/Cost of Illness/Resource Use Studies
Disease
Infectious Disease (non-vaccine), Pediatrics, Systemic Disorders/Conditions (Anesthesia, Auto-Immune Disorders (n.e.c.), Hematological Disorders (non-oncologic), Pain)