Table 2.
Subject of included articles
| Included studies | Effectiveness | Cost-effectiveness | Budget impact | |
|---|---|---|---|---|
| ALL |
8 |
7 |
1 |
1 |
| CAPS |
7 |
7 |
0 |
0 |
| CLL |
3 |
2 |
1 |
0 |
| Fabry (agalsidase α) |
65 |
63 |
0 |
2 |
| Fabry (agalsidase β) |
66 |
64 |
1 |
1 |
| MPSI |
26 |
26 |
0 |
0 |
| MPSII |
22 |
21 |
1 |
1 |
| MPSVI |
24 |
24 |
0 |
0 |
| PNH |
23 |
21 |
1 |
2 |
| Pompe |
53 |
53 |
0 |
0 |
| STS |
41 |
38 |
3 |
1 |
| Total | 338 | 326 | 8 | 8 |
Six studies described both cost-effectiveness and budget impact; Six studies were included both for agalsidase α and agalsidase β; Six studies were included for multiple MPS diseases; In ten articles (one for CAPS; four for Fabry (α); one for Fabry (β); one for MPSI and three for Pompe) more than one study was described.