From: Characterizing expanded access and compassionate use programs for experimental drugs
Characteristics | No. of programs (%) |
---|---|
Sponsor: individual or collaborative sponsor (N = 398 programs) | |
Industry | 241 (60.6) |
NIH | 11 (2.7) |
U.S. Fed | 6 (1.5) |
Othera | 143 (36) |
Interventions provided (N = 398 programs) | |
Drug | 282 (71) |
Biological | 43 (11) |
Device | 40 (10) |
Other | 31 (8) |
Missing data | 2 (.5) |
Conditions treatedb (N = 460) | |
HIV | 26 (6.5) |
Leukemia | 22 (5.5) |
Multiple myeloma | 14 (3.5) |
Cholestasis | 12 (3) |
Melanoma | 11 (2.7) |
Diabetes | 11 (2.7) |
Lymphoma | 9 (2.2) |
Neuroblastoma | 7 (1.7) |
Age group (N = 398 programs) | |
Adult only | 20 (5) |
Adult/senior | 215 (54) |
Child only | 25 (6.3) |
Adult|child | 26 (6.5) |
Child|adult|senior | 111 (27.9) |
Senior only | 1 (.3) |
Single vs. multi-patient program (N = 398 programs) | |
Single-patient program | 3 (.75) |
Multiple patient program | 73 (18.3) |
Missing data | 322 (80.9) |
Report results (N = 398 programs) | |
Results reported | 8 (2%) |
No results reported | 390 (98) |
FDA approval rates of provided drugs (N = 282 programs providing drugs) | |
Drug was FDA approved | 192 (68) |
Drug was not approved | 76 (27) |
Insufficient information or uncertain | 14 (5) |