Table 1

Characteristics of 69 novel drugs approved by both the FDA and EMA between 2005 and 2010 (excluding everolimus and temsirolimus)

Characteristicsn (%)
Agent type
 Small molecule51 (73.9)
 Biological18 (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 metabolism10 (14.5)
 Anti-infectives for systemic use12 (17.4)
 Antineoplastic and immunomodulating agents20 (29.0)
 Blood and blood forming organs5 (7.2)
 Cardiovascular system5 (7.2)
 Nervous system6 (8.7)
 Other*11 (15.9)
Degree of novelty (according to Lanthier et al 19)
 First-in-class24 (34.8)
 Advance-in-class24 (34.8)
 Addition-to-class21 (30.4)
Size of the marketing- authorisation holder
 Large pharmaceutical company44 (63.8)
 Intermediated-size company23 (33.3)
 Small-size and medium-size company2 (2.9)
Premarket evidence
Total number of included patients
 Minimum /maximum18/18 040
 Median (Q1–Q3)923 (324–1996)
 Mean (SD)1806 (2897)
Expected length of treatment
 Acute8 (11.6)
 Intermediate14 (20.3)
 Chronic47 (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.