Background
Methods
Health state transition
Cost measures
Effect measures
Discounting
Sensitivity analyses
Parameter | Mean input value | Standard deviation | Distribution |
---|---|---|---|
Age at Therapy Start | 51.1 | 5.3 | Normal |
Health State Costs ‘Visual Impaired’ | €3,680 | €368 | Normal |
Health State Costs ‘Blind’ | €4,091 | €409 | Normal |
Health State Costs ‘Death’ | €0 | €0 | None |
AVD Costs, first year (Base Case) | €85,000 | €8,500 | Normal |
AVD Costs, first year (Scenario 1) | €55,000 | €5,500 | Normal |
AVD Costs, first year (Scenario 2) | €70,000 | €7,000 | Normal |
AVD Costs, first year (Scenario 3) | €100,000 | €10,000 | Normal |
AVD Costs, first year (Scenario 4) | €115,000 | €11,500 | Normal |
AVD Costs, annual follow-up | €1,500 | €150 | Normal |
Health State Utility ‘Visual Impaired’ | 0.77 | 0,077 | Normal |
Health State Utility ‘Blind’ | 0.61 | 0.061 | Normal |
Health State Utility ‘Death’ | 0 | 0 | None |
Responder Rate AVD (Base Case) | 62.5% | None | None |
Responder Rate AVD (Scenario 1) | 25% | None | None |
Responder Rate AVD (Scenario 2) | 50% | None | None |
Responder Rate AVD (Scenario 3) | 75% | None | None |
Responder Rate AVD (Scenario 4) | 100% | None | None |
Discounting of Costs | 3% | None | None |
Discounting of Effects | 3% | None | None |
Model simulations, model outcome and time horizon
Willigness-to-pay
Results
Base case analyses results
Costs in Euro | AVD | BSC | Incremental |
---|---|---|---|
Mean Costs in Euro | €189,887 | €81,962 | €107,925 |
Standard Deviation | €16,732 | €12,138 | €15,035 |
2.5% Confidence Interval | €155,538 | €57,298 | €77,379 |
97.5% Confidence Interval | €221,407 | €106,697 | €136,992 |
Effect in QALYs
|
AVD
|
BSC
|
Incremental
|
Mean QALYs | 14.19 | 12.16 | 2.03 |
Standard Deviation | 2.12 | 1.80 | 1.94 |
2.5% Confidence Interval | 9.86 | 8.54 | −1.70 |
97.5% Confidence Interval | 18.48 | 15.66 | 5.87 |
Cost-Effectiveness
|
Cost per QALY gained (AVD versus BSC)
| ||
Cost per QALY gained in Euro | €53,165 | ||
Standard Deviation | €13,273,467 | ||
2.5% Confidence Interval | -€501,604 | ||
97.5% Confidence Interval | €757,628 |
Probabilistic sensitivity analyses results
Deterministic sensitivity analyses
Responder rate and AVD cost scenario analyses
Outcome | RR 25% | RR 50% | RR 62.5% | RR 75% | RR 100% | Scenarios |
---|---|---|---|---|---|---|
IC (Euro) | 81,640 | 78,606 | 77,674 | 76,658 | 75,237 | AVD Cost €55,000 |
IE (QALYs) | 0.80 | 1.59 | 2.03 | 2.41 | 3.20 | |
ICER |
102,050
|
49,438
|
38,263
|
31,808
|
23,512
| |
IC (Euro) | 96,276 | 94,752 | 92,826 | 91,983 | 90,158 | AVD Cost €70,000 |
IE (QALYs) | 0.80 | 1.59 | 2.03 | 2.41 | 3.20 | |
ICER |
120,345
|
59,592
|
45,727
|
38,167
|
28,174
| |
IC (Euro) | 110,984 | 109,218 | 107,925 | 106,731 | 104,685 | AVD Cost €85,000 |
IE (QALYs) | 0.80 | 1.59 | 2.03 | 2.41 | 3.20 | |
ICER |
138,730
|
68,691
|
53,165
|
44,287
|
32,714
| |
IC (Euro) | 126,384 | 123,346 | 122,218 | 121,913 | 119,396 | AVD Cost €100,000 |
IE (QALYs) | 0.80 | 1.59 | 2.03 | 2.41 | 3.20 | |
ICER |
157,980
|
77,576
|
60,206
|
50,586
|
37,311
| |
IC (Euro) | 141,566 | 139,301 | 138,746 | 137,416 | 134,948 | AVD Cost €115,000 |
IE (QALYs) | 0.80 | 1.59 | 2.03 | 2.41 | 3.20 | |
ICER |
176,958
|
87,611
|
68,348
|
57,019
|
42,171
|