Table 1. Characteristics of Investigational Drug Cohort.
| Characteristic | Total drugs, No. (% of total) (n = 120) | FDA-approved drugs, No. (% by drug class) (n = 13) |
|---|---|---|
| Drug status at 8 y | ||
| FDA approved | 13 (10.8) | NA |
| Not FDA approved | 107 (89.2) | NA |
| Drug classa | ||
| Immunotherapy drugs | 20 (16.7) | 4 (20.0) |
| Targeted drugs | 84 (70.0) | 8 (9.5) |
| Cytotoxic drugs | 9 (7.5) | 1 (11.1) |
| Other | 7 (5.8) | 0 (0.0) |
| Enrichmentb | ||
| Early enriched | 29 (24.2) | 6 (20.7) |
| Not early enriched | 91 (75.8) | 7 (7.7) |
| Sponsorc | ||
| Large pharmaceutical company | 31 (25.8) | 6 (19.4) |
| Other | 89 (74.2) | 7 (7.9) |
| Noveltyd | ||
| Novel | 71 (59.2) | 5 (7.0) |
| Not novel or NA | 49 (40.8) | 8 (16.3) |
| Orphan drug designatione | ||
| Orphan drug | 38 (31.7) | 11 (28.9) |
| Nonorphan drug | 82 (68.3) | 2 (2.4) |
Abbreviations: FDA, Food and Drug Administration; NA, not applicable.
a
See eMethods 3 in the Supplement.
b
See eMethods 4 in the Supplement.
c
A drug was considered launched by a large pharmaceutical company if 1 of its first 2 efficacy trials was sponsored by a top 15 company by revenue in the year of trial launch.
d
See eMethods 5 in the Supplement.
e
See eMethods 7 in the Supplement.