Table 1.
Characteristics of oncology approvals reviewed in this article.
| Oncology NDA-NMEs and BLAs without PRO information in the medical review documents (N = 22) | Oncology NDA-NMEs and BLAs with PRO information in the medical review documents (N = 45) | p | |
|---|---|---|---|
| Application type | |||
| NDA-NME (n = 45) | 12 (26.7%) | 33 (73.3%) | 0.124 |
| BLA (n = 22) | 10 (45.5%) | 12 (54.5%) | |
| Orphan status | |||
| Orphan (n = 47) | 19 (40.4%) | 28 (59.6%) | 0.043 |
| Non-Orphan (n = 20) | 3 (15.0%) | 17 (75.0%) | |
| Recommendation on regulatory action | |||
| AA (n = 34) | 9 (26.5%) | 25 (73.5%) | 0.260 |
| RA (n = 33) | 13 (39.4%) | 20 (60.6%) | |
| Indication | |||
| Solid tumours (n = 40) | 7 (17.5%) | 33 (82.5%) | 0.001 |
| Haematological malignancies (n = 27) | 15 (55.6%) | 12 (44.4%) |
Abbreviations: BLA, biologic license application; NDA, new drug application; NME, new molecular entity; PRO, patient-reported outcome; RA, regular approval; AA, accelerated approval.