Scenario | YFEOD | Costs | ICER |
---|
| ERT | no ERT | Δ | ERT | no ERT | Δ | |
---|
Base case | 61.70 | 48.90 | 12.80 | €5,716,473 | €171,780 | €5,544,693 | €434,416 |
|
37.77
|
31.97
|
5.80
| €1,206,933 | €50,048 | €1,156,885 | €199,559 |
Production loss included | 61.70 | 48.90 | 12.80 | €5,772,897 | €294,226 | €5,478,670 | €429,243 |
|
37.77
|
31.97
|
5.80
| €1,216,954 | €71,956 | €1,144,998 | €197,508 |
25% ERT costs reduction | 61.70 | 48.90 | 12.80 | €4,338,430 | €171,780 | €4,166,649 | €326,449 |
|
37.77
|
31.97
|
5.80
| €918,801 | €50,048 | €868,753 | €149,857 |
Historical I* | 19.72 | 17.90 | 1.82 | €5,202,872 | €109,342 | €5,093,530 | €2,803,382 |
|
14.83
|
13.70
|
1.14
| €2,391,020 | €54,143 | €2,336,877 | €2,057,891 |
Historical II* | 12.10 | 10.14 | 1.96 | €6,198,258 | €112,774 | €6,085,484 | €3,111,478 |
|
9.27
|
8.01
|
1.26
| €3,031,062 | €59,253 | €2,971,809 | €2,363,665 |
- YFEOD: years free of end organ damage, ICER: incremental cost-effectiveness ratio. *The historical scenarios were based on the patient distribution in the Netherlands when ERT was introduced in April 1991 and were derived from medical histories of patients referred to the AMC until September 2011. The first historical scenario includes the asymptomatic stage, the second historical scenario excludes the asymptomatic stage. While running the historical scenarios, two model adjustments were made: 1) the probability of death was adjusted for the mean age of 36 (Historical I) or 38 (Historical II) years for patients in the cohort at the time of ERT market introduction in the Netherlands; 2) the time horizon too was adjusted for the mean age of the cohort in 1991 and became 49 (Historical I) or 47 (Historical II) years instead of 85.
- €: amount of money in euros.
- Δ: difference between between the scenarios with versus without ERT.