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.

Hamers and Rumeau-Pichon

Hamers and Rumeau-Pichon BMC Pediatrics 2012 12:60   doi:10.1186/1471-2431-12-60

Open Data