Table 3 One-way sensitivity analyses of the cost-effectiveness of introducing MCADD screening and of switching to MS/MS technology for PKU screening
Parameter | Value or range for sensitivity analyses | ICER (€/QALY) |
|---|---|---|
Base-case values | 7 581 | |
MCADD birth prevalence | 1/10 000 to 1/25 000 | 3 444 to 15 856 |
MCADD screening test specificity | 0.9997 to 1 | 7 878 to 6 987 |
Risk of developing a metabolic crisis | 0.75 | 5 881 |
Risk of death within 72 hours of life | 0.05 | 5 902 |
Risk of death after a metabolic crisis | 0.01 to 0.03 | 13 180 to 5 314 |
Risk of mild neurological sequelae | 0 | 9 175 |
Risk of severe neurological sequelae | 0 | 12 823 |
Screening effectiveness (reduction in the risk of developing a metabolic crisis) | 0.5 | 14 351 |
Utility of persons unaffected by MCADD | 0.9 | 8 769 |
Utility of persons with severe neurological sequelae | 0.45 | 7 121 |
Utility of persons with mild neurological sequelae | 0.92 | 7 632 |
Cost of the MCADD screening test (€) | 3.38 to 5.16 | 5 384 to 15 655 |
Annual cost of management of severe | 15 000 to 150 000 | 8 832 to −19 139* |
Annual cost of management of mild neurological sequelae (€) | 4 500 to 120 000 | 7 911 to −17 353* |
Cost of treatment of a metabolic crisis | 4 730 | 7 211 |
% patients receiving L-carnitine supplementation until 18 years of age | 0% to 100% | 6 617 to 8 546 |
Number of medical consultations per year until 6 years of age | 5 | 7 667 |
No discounting | −514* | |
Annual discounting rate | 3% to 6% | 4 954 to 13 598 |