Table 3 Study characteristics of interventional studies
From: Systematic review of available evidence on 11 high-priced inpatient orphan drugs
| Â | Number (% of all effectiveness studies) | Control group | Random-ization | Open label | RCT* | Single center | International | Mean follow-up duration in weeks [min-max] | Mean number of patients [min-max] |
|---|---|---|---|---|---|---|---|---|---|
ALL | 2 (29%) | 0 | 0 | 2 | 0 | 1 | 0 | Unknown | 39 [17-61] |
CAPS | 2 (22%) | 1 | 1 | 1 | 1 | 0 | 2 | 64 [24-104] | 99 [31-166] |
CLL | 2 (100%) | 0 | 0 | 2 | 0 | 0 | 2 | 78 [52-104] | 86 [33-138] |
Fabry (α) | 15 (22%) | 8 | 8 | 7 | 6 | 4 | 5 | 35 [10-104] | 26 [10-80] |
Fabry (β) | 10 (16%) | 4 | 3 | 8 | 2 | 2 | 5 | 85 [20-234] | 50 [13-134] |
MPSI | 3 (12%) | 2 | 2 | 2 | 1 | 0 | 3 | 35 [26-52] | 33 [20-45] |
MPSII | 3 (14%) | 2 | 2 | 1 | 2 | 0 | 1 | 44 [26-53] | 39 [10-96] |
MPSVI | 3 (13%) | 0 | 1 | 2 | 0 | 0 | 2 | 95 [48-190] | 8 [7-10] |
PNH | 4 (19%) | 1 | 1 | 3 | 1 | 1 | 2 | 26 [12-52] | 56 [11-97] |
Pompe | 8 (14%) | 3 | 2 | 7 | 1 | 3 | 4 | 70 [26-120] | 23 [5-90] |
STS | 12 (32%) | 0 | 1 | 12 | 0 | 0 | 6 | 42 [9-104] | 64 [13-270] |
Total | 64 (19%) | 21 | 21 | 47 | 14 | 11 | 32 | 57 [9-234] | 48 [7-270] |