Table 2.
Characteristics of the study sample
| Characteristics | EMA-approved cancer indications (n = 144), n (%) |
|---|---|
| Indication (ICD-10 category) | |
| Cancer of lip, oral cavity and pharynx (C00-C14) | 1 (0.7) |
| Gastrointestinal cancer (C15-C26) | 22 (15.3) |
| Lung cancer (C30-C39) | 34 (23.6) |
| Melanoma (C43-C44) | 21 (14.6) |
| Sarcoma (C45-C49) | 2 (1.4) |
| Breast cancer (C50-C50) | 21 (14.6) |
| Cervical carcinoma (C51-C58) | 5 (3.5) |
| Ovarian and peritoneal cancer (C51-C58 and C45-C49) | 5 (3.5) |
| Prostate cancer (C60-C63) | 12 (8.3) |
| Renal cell carcinoma (C64-C68) | 14 (9.7) |
| Thyroid carcinoma and neuroendocrine tumour (C73-C75) | 4 (2.8) |
| Cancer of ill-defined, secondary and unspecified sites (C76-C80) | 3 (2.1) |
| Phase of the study | |
| II | 6 (4.2) |
| II/III | 1 (0.7) |
| III | 137 (95.1) |
| Study participants, n | |
| Mean | 707 |
| Median | 581 |
| Range, n | 50-4805 |
| Statistically significant study endpoints | |
| OS | 65 (45.1) |
| PFS | 111 (77.1) |
| Other | 13 (9.0) |
| ESMO-MCBS forms (original and adapted) | |
| Curative | |
| 1 | 9 (6.3) |
| Non-curative | |
| 2a | 56 (38.9) |
| 2b | 72 (50.0) |
| 2c | 7 (4.9) |
| ESMO-MCBS adjustments | |
| Original (n = 36) | |
| +1 | 31 (86.1) |
| −1 | 3 (8.3) |
| +1/−1 | 2 (5.6) |
| Adapted (n = 95) | |
| +1 | 21 (22.1) |
| +2 | 3 (3.2) |
| −1 | 61 (64.2) |
| −2 | 1 (1.1) |
| +1/−1 | 9 (9.5) |
| Approval year | |
| 2009 | 9 (6.3) |
| 2010 | 9 (6.3) |
| 2011 | 11 (7.6) |
| 2012 | 6 (4.2) |
| 2013 | 11 (7.6) |
| 2014 | 11 (7.6) |
| 2015 | 15 (10.4) |
| 2016 | 18 (12.5) |
| 2017 | 14 (9.7) |
| 2018 | 17 (11.8) |
| 2019 | 16 (11.1) |
| 2020 (until Oct) | 7 (4.9) |
| EMA authorisation route/label | |
| Regular EMA approval | 35 (24.3) |
| Additional monitoring (AM) | 92 (63.9) |
| Conditional approval (CA) | 13 (9.0) |
| Orphan designation (OD) | 25 (17.4) |
| AM and CA | 4 (2.8) |
| AM and OD | 12 (8.3) |
| CA and OD | 3 (2.1) |
| AM and OD and CA | 1 (0.7) |
Deviation of 100% cumulative percentage may be caused by rounding.
AM, additional monitoring; CA, conditional approval; EMA, European Medicines Agency; EMSO, European Society for Medical Oncology; ICD-10, International Classification of Disease (10th Revision); n, number of solid cancer indications; Magnitude of Clinical Benefit Scale; OD, orphan designation; OS, overall survival; PFS, progression-free survival.