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Table 1 Characteristics of registered expanded access and compassionate use programs

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)
  1. aGenerally, academic institutions
  2. bTop 8 conditions with the most programs