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)