Appendix 2Summary of Key Outcomes
Table 5When Considering Only Costs, Outcomes & Quality of Life, How Attractive Is Migalastat Relative to Agalsidase alfa?
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Migalastat vs. Agalsidase alfa | Attractive | Slightly Attractive | Equally Attractive | Slightly Unattractive | Unattractive | NA |
---|
Costs (total) | | | | ✕ | | |
Drug treatment costs alone | | | | ✕ | | |
Clinical outcomes | | | ✕ | | | |
Quality of life | | | ✕ | | | |
ICER or net benefit calculation | CDR revised base case: the ICUR for migalastat vs. agalsidase alfa ranges from $200,487 per QALY to $55,935,921 per QALY. CDR noted that there is a substantial uncertainty around the efficacy equivalence and the disutility value assumptions. |
CDR = CADTH Common Drug Review; ICER = incremental cost-effectiveness ratio; ICUR = incremental cost-utility ratio; QALY = quality-adjusted life-year; vs. = versus.
Table 6When Considering Only Costs, Outcomes & Quality of Life, How Attractive Is Migalastat Relative to Agalsidase beta?
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Migalastat vs. Agalsidase beta | Attractive | Slightly Attractive | Equally Attractive | Slightly Unattractive | Unattractive | NA |
---|
Costs (total) | | ✕ | | | | |
Drug treatment costs alone | | ✕ | | | | |
Clinical outcomes | | | ✕ | | | |
Quality of life | | | ✕ | | | |
ICER or net benefit calculation | CDR revised base case: migalastat is dominant compared with agalsidase beta. CDR noted that there is a substantial uncertainty around the efficacy equivalence and the disutility value assumptions. |
CDR = CADTH Common Drug Review; ICER = incremental cost-effectiveness ratio; vs. = versus.