|
2008–2012
(n = 7)
|
2013–2016
(n = 13)
|
2017–2020
(n = 21)
|
Total
(n = 41)
|
P valueb
|
Pivotal trial, n(%) |
|
|
|
|
0.6005 |
Yes |
6 (85.71) |
11 (84.62) |
15 (71.43) |
32 (78.05) |
|
No |
1 (14.29) |
2 (15.38) |
6 (28.57) |
9 (21.95) |
|
Design types, n(%) |
|
|
|
|
0.7568 |
Seamless design |
3 (42.86) |
5 (38.46) |
7 (33.33) |
15 (36.59) |
|
Group sequential |
2 (28.57) |
5 (38.46) |
10 (47.62) |
17 (41.46) |
|
Sample size re-estimation |
1 (14.29) |
0 (0.00) |
0 (0.00) |
1 (2.44) |
|
Combined groupa
|
0 (0.00) |
1 (7.69) |
0 (0.00) |
1
(2.44) |
|
Adaptations to treatment arm selection |
0 (0.00) |
0 (0.00) |
1 (4.76) |
1 (2.44) |
|
Adaptive dose-ranging |
0 (0.00) |
0 (0.00) |
1 (4.76) |
1 (2.44) |
|
Basket study |
0 (0.00) |
0 (0.00) |
1 (4.76) |
1 (2.44) |
|
Enrichment design |
1 (14.29) |
2 (15.38) |
1 (4.76) |
4 (9.76) |
|
Orphan drug, n(%) |
|
|
|
|
0.5069 |
Yes |
2 (28.57) |
3 (23.08) |
9 (42.86) |
14 (34.15) |
|
No |
5 (71.43) |
10 (76.92) |
12 (57.14) |
27 (65.85) |
|
Antitumor drug, n(%) |
|
|
|
|
1.0000 |
Yes |
3 (42.86) |
6 (46.15) |
10 (47.62) |
19 (46.34) |
|
No |
4 (57.14) |
7 (53.85) |
11 (52.38) |
22 (53.66) |
|
Accelerated assessment, n(%) |
|
|
|
|
0.4110 |
Yes |
1 (14.29) |
0 (0.00) |
2 (9.52) |
3 (7.32) |
|
No |
6 (85.71) |
13 (100.00) |
19 (90.48) |
38 (92.68) |
|
New active substance, n(%) |
|
|
|
|
0.0912 |
Yes |
4 (57.14) |
9 (69.23) |
19 (90.48) |
32 (78.05) |
|
No |
3 (42.86) |
4 (30.77) |
2 (9.52) |
9 (21.95) |
|
Additional monitoring, n(%) |
|
|
|
|
<0.0001 |
Yes |
0 (0.00) |
3 (23.08) |
19 (90.48) |
22 (53.66) |
|
No |
7 (100.00) |
10 (76.92) |
2 (9.52) |
19 (46.34) |
|
CMA, PASS, PAES n(%)c |
|
|
|
|
0.3286 |
Yes |
3 (42.86) |
3 (23.08) |
11 (52.38) |
17 (41.46) |
|
No |
4 (57.14) |
10 (76.92) |
10 (47.62) |
24 (58.54) |
|
System organ class indications, n(%) |
|
|
|
|
0.0578 |
Nervous system disorders |
0 (0.00) |
1 (7.69) |
0 (0.00) |
1 (2.44) |
|
Congenital, familial, and genetic disorders |
1 (14.29) |
0 (0.00) |
1
(4.76) |
2 (4.88) |
|
Renal and urinary disorder |
2 (28.57) |
0 (0.00) |
0 (0.00) |
2 (4.88) |
|
Respiratory, thoracic, and mediastinal disorders |
3 (42.86) |
1 (7.69) |
0 (0.00) |
4 (9.76) |
|
Neoplasms benign, malignant, and
unspecified (incl. cysts and polyps) |
1 (14.29) |
1 (7.69) |
5 (23.81) |
7 (18.92) |
|
Blood and lymphatic system disorders |
0 (0.00) |
3 (23.08) |
5 (23.81) |
8 (19.51) |
|
Gastrointestinal disorders |
0 (0.00) |
1 (7.69) |
3 (14.29) |
4 (8.11) |
|
Skin and subcutaneous tissue disorders |
0 (0.00) |
1 (7.69) |
2 (9.52) |
3 (7.32) |
|
Immune system disorders |
0 (0.00) |
0 (0.00) |
2 (9.52) |
2 (4.88) |
|
Vascular disorders |
0 (0.00) |
2 (15.38) |
0 (0.00) |
2 (4.88) |
|
Metabolism and nutrition disorders |
0 (0.00) |
1 (7.69) |
0 (0.00) |
1
(2.44) |
|
Cardiac disorders |
0 (0.00) |
1 (7.69) |
0 (0.00) |
1 (2.44) |
|
Infections and infestations |
0 (0.00) |
1 (7.69) |
1 (4.76) |
2 (4.88) |
|
Psychiatric disorders |
0 (0.00) |
0 (0.00) |
1 (4.76) |
1 (2.44) |
|
Musculoskeletal and connective tissue disorders |
0 (0.00) |
0 (0.00) |
1 (4.76) |
1 (2.44) |
|