Table 4 Study characteristics of observational studies and case studies
From: Systematic review of available evidence on 11 high-priced inpatient orphan drugs
| Â | Observational studies | Case studies | ||||
|---|---|---|---|---|---|---|
| Â | Number (% of all effectiveness studies) | Control group | Single center | Registry data | Mean number of patients [min-max] | Number (% of all effectiveness studies) |
ALL | 1 (14%) | 0 | 0 | 0 | 5 [5] | 4 (57%) |
CAPS | 5 (56%) | 3 | 2 | 0 | 25 [10-35] | 2 (22%) |
CLL | 0 (0%) | - | - | - | - | 0 (0%) |
Fabry (α) | 37 (55%) | 10 | 17 | 13 | 102 [7-752] | 15 (22%) |
Fabry (β) | 29 (45%) | 13 | 18 | 3 | 62 [6-822] | 25 (39%) |
MPSI | 7 (27%) | 0 | 5 | 1 | 141 [5-891] | 16 (62%) |
MPSII | 9 (41%) | 1 | 3 | 3 | 56 [11-124] | 10 (45%) |
MPSVI | 5 (21%) | 0 | 1 | 1 | 41 [3-132] | 16 (67%) |
PNH | 8 (38%) | 5 | 0 | 0 | 58 [6-187] | 9 (43%) |
Pompe | 14 (25%) | 2 | 4 | 0 | 26 [8-74] | 34 (61%) |
STS | 17 (45%) | 3 | 8 | 0 | 60 [7-379] | 9 (24%) |
Total | 132 (39%) | 37 | 58 | 21 | 58 [3-891] | 140 (42%) |