Drug approved with ongoing studyb (n = 17) Drug approved with completed studyc (n = 24) Total (n = 41)
P valued
Pivotal trial, n(%) 0.7113
Yes 14 (82.35) 18 (75.00) 32 (78.05)
No 3 (17.65) 6 (25.00) 9 (21.95)
Design types, n(%) 0.1932
Seamless design 6 (35.29) 9 (37.50) 15 (36.59)
Group sequential 9 (52.94) 8 (33.33) 17 (41.46)
Sample size re-estimation 1 (5.88) 0 (0.00) 1 (2.44)
Combined groupa 0 (0.00) 1 (4.17) 1 (2.44)
Adaptations to treatment arm selection 0 (0.00) 1 (4.17) 1 (2.44)
Adaptive dose-ranging 0 (0.00) 1 (4.17) 1 (2.44)
Basket study 1 (5.88) 0 (0.00) 1 (2.44)
Enrichment design 0 (0.00) 4 (16.67) 4 (9.76)
Orphan drug, n(%) 0.1887
Yes 8 (47.06) 6 (25.00) 14 (34.15)
No 9 (52.94) 18 (75.00) 27 (65.85)
Antitumor drug, n(%) 0.0001
Yes 14 (82.35) 5 (20.83) 19 (46.34)
No 3 (17.65) 19 (79.17) 22 (53.66)
Accelerated assessment, n(%) 1.0000
Yes 1 (5.88) 2 (8.33) 3 (7.32)
No 16 (94.12) 22 (91.67) 38 (92.68)
New active substance, n(%) 0.2623
Yes 15 (88.24) 17 (70.83) 32 (78.05)
No 2 (11.76) 7 (29.17) 9 (21.95)
Additional monitoring, n(%) 0.1120
Yes 12 (70.59) 10 (41.67) 22 (53.66)
No 5 (29.41) 14 (58.33) 19 (46.34)
CMA, PASS, PAES n(%)e 0.0230
Yes 11 (64.71) 6 (25.00) 17 (41.46)
No 6 (35.29) 18 (75.00) 24 (58.54)
System organ class of indications, n(%) 0.1201
Nervous system disorders 0 (0.00) 1 (4.17) 1 (2.44)
Congenital, familial, and genetic disorders 1 (5.88) 1 (4.17) 2 (4.88)
Renal and urinary disorder 1 (5.88) 1 (4.17) 2 (4.88)
Respiratory, thoracic, and mediastinal disorders 1 (5.88) 3 (12.50) 4 (9.76)
Neoplasms benign, malignant and unspecified (incl cysts and polyps) 6 (35.29) 1 (4.17) 7 (18.92)
Blood and lymphatic system disorders 5 (29.41) 3 (12.50) 8 (19.51)
Gastrointestinal disorders 1 (5.88) 3 (12.50) 4 (8.11)
Skin and subcutaneous tissue disorders 0 (0.00) 3 (12.50) 3 (7.32)
Immune system disorders 0 (0.00) 2 (8.33) 2 (4.88)
Vascular disorders 0 (0.00) 2 (8.33) 2 (4.88)
Metabolism and nutrition disorders 0 (0.00) 1 (4.17) 1 (2.44)
Cardiac disorders 0 (0.00) 1 (4.17) 1 (2.44)
Infections and infestations 2 (11.76) 0 (0.00) 2 (4.88)
Psychiatric disorders 0 (0.00) 1 (4.17) 1 (2.44)
Musculoskeletal and connective tissue disorders 0 (0.00) 1 (4.17) 1 (2.44)