Table 1.
Characteristics of 69 novel drugs approved by both the FDA and EMA between 2005 and 2010 (excluding everolimus and temsirolimus)
| Characteristics | n (%) |
| Agent type | |
| Small molecule | 51 (73.9) |
| Biological | 18 (26.1) |
| Orphan status (FDA) | 18 (26.1) |
| Orphan designation (EMA) | 20 (29.0) |
| Accelerated approval (FDA) | 14 (20.3) |
| Therapeutic class according to the ATC classification | |
| Alimentary tract and metabolism | 10 (14.5) |
| Anti-infectives for systemic use | 12 (17.4) |
| Antineoplastic and immunomodulating agents | 20 (29.0) |
| Blood and blood forming organs | 5 (7.2) |
| Cardiovascular system | 5 (7.2) |
| Nervous system | 6 (8.7) |
| Other* | 11 (15.9) |
| Degree of novelty (according to Lanthier et al19) | |
| First-in-class | 24 (34.8) |
| Advance-in-class | 24 (34.8) |
| Addition-to-class | 21 (30.4) |
| Size of the marketing- authorisation holder | |
| Large pharmaceutical company | 44 (63.8) |
| Intermediated-size company | 23 (33.3) |
| Small-size and medium-size company | 2 (2.9) |
| Premarket evidence | |
| Total number of included patients | |
| Minimum /maximum | 18/18 040 |
| Median (Q1–Q3) | 923 (324–1996) |
| Mean (SD) | 1806 (2897) |
| Expected length of treatment | |
| Acute | 8 (11.6) |
| Intermediate | 14 (20.3) |
| Chronic | 47 (68.1) |
*Includes dermatological, genitourinary system and sex hormones, musculoskeletal system, sensory organs, systemic hormonal preparations, excluding sex hormones, and others.
ATC, anatomical therapeutic chemical; EMA, European Medicines Agency; FDA, Food and Drug Administration.