Characteristics of publications of confirmatory studies
| Indication | All publications | Randomised trial publications | Outcome used in all publications | Number of patients per study/trial (median, IQR) | ||||
| Total number | Number observational (%) | Number randomised (%) | Number blinded (%) | Number with active controls (%) | Number surrogate (%) | Number clinical (%) | ||
| Oncology | 20 | 2 (10) | 18 (90) | 5* (27.8) | 14 (77.8) | 11 (55) | 9 (45) | 710 (270–1636) |
| HIV/AIDS | 5 | 0 (0) | 5 (100) | 2† (40) | 5 (100) | 5 (100) | 0 (0) | 562 (130–584) |
| Other‡ | 11 | 5 (45.5) | 6 (54.5) | 3 (50) | 2§§ (33.3) | 4 (36.4) | 7 (63.6) | 103 (41–671) |
| Total | 36 | 7 (19.4) | 29 (80.6) | 10 (34.5) | 21 (72.4) | 20 (55.6) | 16 (44.4) | 541 (122–877) |
*Five did not state if blinded.
†One stated ‘partially blinded’.
‡Central neuropathic pain, clotting disorders, hepatitis C, influenza, Parkinson’s disease, paroxysmal nocturnal haemoglobinuria, stroke.
↵§One randomised to two different durations of treatment.