Table 1.
Characteristics of the 228 ClinicalTrials.gov-registered, expanded access studies.
| Characteristics | Number of studies, n (%) | |
| Medical specialty |
|
|
|
|
Oncology | 96 (42.1) |
|
|
Neurology | 29 (12.7) |
|
|
Infectious diseases | 28 (12.3) |
|
|
Hematology | 16 (7.0) |
|
|
Metabolic diseases | 15 (6.6) |
|
|
Gastroenterology | 8 (3.5) |
|
|
Cardiovascular diseases | 7 (3.1) |
|
|
Pulmonology | 5 (2.2) |
|
|
Other | 17 (7.5) |
|
|
Unknown | 7 (3.1) |
| Intervention type |
|
|
|
|
Drug | 162 (71.1) |
|
|
Biologic | 61 (26.8) |
|
|
Medical device | 5 (2.2) |
| Sponsor |
|
|
|
|
Industry | 182 (79.8) |
|
|
Nonindustry | 32 (14.0) |
|
|
Mixed | 14 (6.1) |
| Center location |
|
|
|
|
United States | 75 (32.9) |
|
|
International | 16 (7.0) |
|
|
Other | 14 (6.1) |
|
|
Unknown | 123 (54.0) |
| Expanded access type |
|
|
|
|
IPa | 103 (45.2) |
|
|
ISb | 44 (19.3) |
|
|
Tr. IND/Pr.c | 46 (20.2) |
|
|
Mixedd | 24 (10.5) |
|
|
Unknown | 11 (4.8) |
| Multicenter studies |
|
|
|
|
Yes | 50 (21.9) |
|
|
No | 55 (24.1) |
|
|
Unknown | 123 (54.0) |
| Multinational studies |
|
|
|
|
Yes | 16 (7.0) |
|
|
No | 91 (39.9) |
|
|
Unknown | 121 (53.1) |
aIP: individual patients.
bIS: intermediate-size population.
cTr. IND/Pr.: treatment investigational new drug (IND)/protocol.
dAny combination of individual patients, intermediate-size population, and treatment IND/protocol.