Table. Characteristics of Accelerated Approval Confirmatory Trial Requirements.
| Drug characteristic | No. clinical trial requirementsa | Median time between approval and deadline (IQR), y | No. trials completed or due by September 15, 2021 | Late trials, No. (%) |
|---|---|---|---|---|
| All | 177 | 3.5 (1.8-5.8) | 100 | 54 (54) |
| Therapeutic areab | ||||
| Genetic disease | 8 | 8.7 (4.6-9.3) | 1 | 1 (100) |
| Infectious disease | 6 | 4.2 (2.0-8.0) | 0 | 0 |
| Neurology | 2 | 8.0 (7.6-8.4) | 1 | 1 (100) |
| Hematology | 15 | 2.5 (1.1-4.0) | 14 | 13 (93) |
| Autoimmune disease | 4 | 6.9 (6.5-6.9) | 0 | 0 |
| Cancer | 142 | 3.5 (1.8-5.6) | 84 | 39 (46) |
| Drug type | ||||
| Small molecule | 110 | 3.7 (1.8-6.1) | 65 | 40 (62) |
| Biologic | 67 | 3.3 (1.8-5.4) | 35 | 14 (40) |
a
We identified accelerated approvals from January 1, 2012 and July 31, 2021 using the CDER Drug and Biologic Accelerated Approval Based on Surrogate Endpoint (https://www.fda.gov/drugs/nda-and-bla-approvals/accelerated-approvals). Postmarket trial requirements and the deadlines for each requirement were identified from correspondence on Drugs@FDA and ranged from 0.1-14.0 years after approval.
b
Therapeutic area was categorized by the authors. Treatment categories with fewer than 5 indications (autoimmune disease and neurology) were combined for analysis. There was a significant variation in time granted by therapeutic area.