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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