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 |
* A negative cost-effectiveness ratio indicates that the strategy is both more effective and less costly than the comparison strategy.