Budget impact analysis for sickle cell disease point-of-care testing in Mozambique Free

Background: Sickle cell disease (SCD) is a leading cause of under-five mortality and morbidity in Sub-Saharan Africa. Effective neonatal screening enables early linkage to care leading to improved outcomes, reduced long-term complications and lower economic costs. Standard diagnostics such as high-performance liquid chromatography (HPLC) require advanced laboratories and skilled staff, limiting scalability in low-resource settings. Point-of-care testing (POCT) offers a cost-effective alternative, enabling rapid diagnosis without complex equipment or extensive training. However, the financial implications of nationwide POCT-based screening programs remain uncertain. Mozambique, an East African low-income country without universal newborn screening, faces a substantial but poorly defined SCD burden. Determining the financial impact of a nationwide POCT-based newborn screening and treatment program is essential for planning and future sustainability.Objective:To estimate the 5-year budgetary impact of a national POCT screening and treatment program for SCD in Mozambique and identify key drivers via sensitivity analysis.

Methods: Annual birth cohorts were projected using United Nations demographic data for Mozambique, applying a 2.8% annual growth rate. In Year 1, universal screening of all children under one-year was assumed. Using Hardy-Weinberg calculations and estimated allele frequencies, SCD prevalence was estimated at 1%, representing those expected to be diagnosed and treated. From Years 2–5, we assumed that all prevalent cases had been identified in Year 1, and only incident cases (0.1% of newborns annually, per 2023 Lancet Global Burden of Disease estimates) entered treatment. Cost inputs included screening (HemoTypeSC™ and Gazelle™ Ferritin Test) confirmatory testing for all positive diagnoses, hydroxyurea therapy, penicillin, folic acid, vaccines, and hospital admissions. Country-specific test and medication costs included freight, insurance, and procurement expenses. Healthcare utilization and cost data were drawn from literature, World Health Organization CHOICE unit cost estimates tool, and Mozambique's Ministry of Health expenditure records. A comparator model using Sickle SCAN® was also analyzed. Outcomes included total annual cost, per-member-per-month (PMPM) cost, and cumulative budget impact over five years. One-way sensitivity analyses varied clinical and economic parameters by ±20%. All analyses performed using Microsoft Excel.

Results: In Year 1 we estimated approximately 12,219 newborns were diagnosed with SCD through the nationwide POCT screening program, incurring total costs of $6.6 million and a PMPM cost of $0.45. By Year 3, total annual costs slightly increased to $6.9 million (PMPM $0.45), reflecting a more stable treatment population and ongoing healthcare needs. Cumulative costs by Year 5 increased to $11.8 million with a PMPM of $0.71, driven by sustained treatment requirements and hospital admissions as the cohort aged. Use of Sickle SCAN®, increased Year 1 costs to $ 15.3 million (PMPM $1.05). Sensitivity analyses identified diagnostic test specificity as the most influential cost driver. Reducing specificity from 100% to 80% resulted in a significant increase in total costs, from $6.7 million to over $300 million, primarily due to the financial burden of false-positive results requiring unnecessary confirmatory testing and treatment. Other significant drivers included the decrease in test sensitivity, hospital admission expenses, and pricing of diagnostic tests. Importantly, existing clinical validation studies demonstrate that all POCT used in the model possess high sensitivity and specificity, generally ranging from 95% to 100%, supporting the validity of our base case assumptions and reinforcing the reliability of the projected budget impact.

Conclusions: A nationwide SCD screening and treatment program using POCT in Mozambique requires significant but manageable financial resources, with stable PMPM costs over time. Ensuring high diagnostic specificity is essential to avoid unnecessary costs from false positives. Long-term investments in early diagnosis and treatment could reduce costly hospitalizations and improve survival and quality of life for children with SCD. Policymakers should consider phased implementation, cost negotiations, reliable diagnostics and international partnerships to enhance affordability and sustainability in LMIC contexts.