|
Studies funded and started
|
39
|
62.9
|
23
|
37.1
| |
62
|
100.0
|
|
Clinical area
| | | | |
0.604
| | |
|
Oncology/hematology
|
11
|
28.2
|
8
|
34.8
| |
19
|
30.6
|
|
Congenital and genetic disorders
|
13
|
33.3
|
4
|
17.4
| |
17
|
27.4
|
|
Immune system disorders
|
4
|
10.3
|
3
|
13.0
| |
7
|
11.3
|
|
Other
|
11
|
28.2
|
8
|
34.8
| |
19
|
30.6
|
|
Prevalence of rare disease
| | | | |
0.381
| | |
|
> 1/10,000
|
14
|
35.9
|
7
|
30.4
| |
21
|
33.9
|
|
1–9/100,000
|
12
|
30.8
|
11
|
47.8
| |
23
|
37.1
|
|
1–9/1,000,000
|
13
|
33.3
|
5
|
21.8
| |
18
|
29.0
|
|
Study characteristics
| | | | | | | |
|
Design
| | | | |
0.055
| | |
|
Randomized clinical trials (RCT)
|
21
|
53.8
|
18
|
78.3
| |
39
|
62.9
|
|
Uncontrolled clinical trials (CT)
|
18
|
46.2
|
5
|
21.7
| |
23
|
37.1
|
|
Type of control group (n = 39)
|
21
|
100.0
|
18
|
100.0
|
0.584
|
39
|
100.0
|
|
Active control
|
11
|
52.4
|
11
|
61.1
| |
22
|
56.4
|
|
Placebo control/no treatment
|
10
|
47.6
|
7
|
38.9
| |
17
|
43.6
|
|
Planned sample size (tertiles)
| | | | |
0.901
| | |
|
< =46 patients
|
13
|
33.3
|
8
|
34.8
| |
21
|
33.9
|
|
47-100 patients
|
14
|
35.9
|
7
|
30.4
| |
21
|
33.9
|
|
> 100 patients
|
12
|
30.8
|
8
|
34.8
| |
20
|
32.3
|
|
Mean
|
98
| |
107
| | |
101
| |
|
Median
|
60
| |
80
| | |
60
| |
|
Q1-Q3
|
30–120
| |
29–177
| | |
30–124
| |
|
Special populations
| | | | |
0.307
| | |
|
Only pediatrics
|
12
|
30.8
|
6
|
26.1
| |
18
|
29.0
|
|
Also pediatrics
|
4
|
10.3
|
0
|
0.0
| |
4
|
6.5
|
|
Only adults
|
15
|
38.5
|
9
|
39.1
| |
24
|
38.7
|
|
Adults and elderly
|
8
|
20.5
|
8
|
34.8
| |
16
|
25.8
|
