Table 1 Included studies
Author publication year | Country/ Perspective sponsored | Currency | Comparison | Treatment | Treatment Incremental Costs | Costs converted to PPP Euro 2022 Netherlands | Time Horizon | Discounting | Model Type | Utility scores (average) | LYG gained | QALY’s gained | ICER | Currency conversion to PPP 2022 Euro (Netherlands) | |
|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
NCPE 2018 | Societal perspective | 2018 Euros | ERT compared to existing therapies | Sebelipase alfa Weight based dose Starting 0.35 mg/kg | N/R | Lifetime | NR | Cost-effectiveness state-transition model | NR | LAL-CL03: 67% 12-month survival LAL-CL02: N/A LAL-1-NH01 (Cohort): 0% > 12 months survival | NR | ICER: € 2,813,000/QALY (infantile cohort) € 2,701,000/QALY (paediatric adult cohort) | ICER: € 2,940,582/QALY (infantile cohort) 2,823,502/ QALY (paediatric adult cohort | ||
Castro Jaramillo 2012 | Colombia NHS perspective No funding | 2012GBP | ERT compared to supportive therapy. (IOPD) | Alglucosidase 20 mg/kg/2 weeks | £ £557,653 | €755,875.60 | 20 years | 5% | Cost-effectiveness micro-simulation model | 0.700 | NR | 5.07 | (ICER): £ 109.991 per QALY earned | ICER 149,088 per QALY earned | |
Castro Jaramillo 2012 | England NHS perspective No funding | 201GBP | ERT compared to supportive therapy. (IOPD) | Alglucosidase 20 mg/kg/2 weeks | £ 1187.940 | €1,610,203 | 20 years | 5% | Cost-effectiveness patient-simulation model | 0.700 | NR | 5.07 | ICER £234.308 per QALY earned | ICER 317,594 | |
Kanters et al. 2014 | Netherlands Societal perspective funded by the Netherlands Organization for Health Research and Development | 2009 Euros | ERT compared to supportive therapy (IOPD) | Alglucosidase 20 mg/kg/2 weeks | €1,9 million | €2,130,300 | Lifetime | 4% cost and 1,5% effect | Cost-effectiveness patient-simulation model | 0.24–0.82 (0.62) | 13.39 | 6,75 | ICER: € 286.114 per QALY gained | 320,794 | |
Kanters et al. 2014 | Netherlands Societal perspective funded by the Netherlands Organization for Health Research and Development | 2009 Euros | ERT compared to Supportive therapy (IOPD) | Alglucosidase 40 mg/kg/weeks | €7,032,899 | €7,885,363 | Lifetime | 4% cost and 1,5% effect | Cost-effectiveness patient-simulation model | 0.24–0.82 (0.62) | 13.39 | 6.75 | ICER €1.043.868 per QALY gained | ICER €1,170,396 per QALY | |
Kanters et al. 2017 | Netherlands Societal perspective funded by the Netherlands Organization for Health Research and Development | 2014 Euros | ERT compared to Supportive therapy (LOPD) | Alglucosidase 20 mg/kg/2 weeks | Sc.1: €6.5 million Sc.2:€7.6 million | Sc 1: €7,117,241.Sc 2:€8,321,697 | Lifetime | 1,5% effects, 4.0% costs | Cost-effectiveness patient-simulation model | 0,45 | Scenario 1: 2.03 Scenario 2: 5.67 | Scenario 1: 2.13 Scenario 2: 4.38 | ICER (SCENARIO 1) €3.167.914 million per QALY gained SCENARIO 2 €1.774.390 million per QALY gained | ICER (SCENARIO 1)€3.468.739 per QALY gained Scenario 2 € 1.942.886 per QALY gained | |
Rombach et. Al. 2013 | Netherlands Societal perspective Funded by a grant from the Ministry of Health | 2009 Euros | ERT compared to Standard medical care (Fabry) | agalsidase alfa or beta | € 2.420.956 | €2,721,258 | Lifetime | 4% cost and 1,5% effect | Cost-effectiveness state-transition model | NR | NR | 0.7 YFEOD:0.7 | ICER €3.318.239 per YFEOD € 3.282.252 per QALY | ICER €3.729.842 per YFEOD ICER € 3.689.391 per QALY | |
Van Dussen et. Al., 2014 | Netherlands Societal perspective Funded by the Dutch Top Institute Pharma project nr T6-208 | 2009 Euros | ERT compared to Standard medical care (Gaucher) | imiglucerase vial of 400UI | € 1.206.933 | €1,353,226 | Lifetime | 4% cost and 1,5% effect | Cost-effectiveness state-transition model | NR | NR | 2.67 YFEOD: 5.8- | ICER €199,559 per YFEOD gained € 432,540 per QALY gained | ICER €223.747 per YFEOD ICER €484.968 per QALY gained |