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Table 4 Study design of the pivotal studies (n = 108)

From: Clinical evidence for orphan medicinal products-a cause for concern?

  Number of pivotal studies (%)
Control arm
No control 34 (31.5%)
Controlled 74 (68.5%)
  Historical control 2 (1.9%)
  Different dosages of the OMP 11 (10.2%)
  Placebo 49 (45.4%)
  Active comparator (or standard of care) 17 (15.7%)
Similarity at baseline
  Yes, statistically verified 13 (12.0%)
  Likely, but not statistically verifiable 41 (38.0%)
  Not likely, but not statistically verifiable 4 (3.7%)
  No, statistically verified 1 (0.9%)
  Not reported 15 (13.9%)
Randomized allocation
No 38 (35.2%)
Yes 70 (64.8%)
  Valid method of randomization 25 (23.1%)
  Invalid method of randomization 2 (1.9%)
  Not reported 43 (39.8%)
Blinding
No (open-label) 44 (40.7%)
No, but justified 10 (9.3%)
Yes 54 (50.0%)
  Blinding of the care provider 53 (49.1%)
  Blinding of the outcomes assessor 12 (11.1%)
  Blinding of the patient 54 (50.0%)