Table 1.
Frequency distribution of specific variables according to the final decision on drug innovativeness
| All drug reports [n = 141] |
Fully innovative [n = 45] |
Conditionally innovative [n = 42] |
Not innovative [n = 54] |
p-value | |
|---|---|---|---|---|---|
| Period of assessment | |||||
| 2017 | 27 (19.2) | 7 (15.6) | 10 (23.8) | 10 (18.5) | 0.48 |
| 2018 | 26 (18.4) | 9 (20.0) | 8 (19.0) | 9 (16.7) | |
| 2019 | 37 (26.2) | 11 (24.4) | 7 (16.7) | 19 (35.2) | |
| 2020 | 35 (24.8) | 11 (24.4) | 14 (33.3) | 10 (18.5) | |
| 2021 | 16 (11.4) | 7 (15.6) | 3 (7.1) | 6 (11.1) | |
| Rare diseasea | |||||
| No | 67 (48.2) | 20 (46.5) | 12 (28.6) | 35 (64.8) | 0.002 |
| Yes | 72 (51.8) | 23 (53.5) | 30 (71.4) | 19 (35.2) | |
| Orphan drug designationb | |||||
| No | 84 (59.6) | 23 (51.1) | 24 (57.1) | 37 (68.5) | 0.20 |
| Yes | 57 (40.4) | 22 (48.9) | 18 (42.9) | 17 (31.5) | |
| Type of disease | |||||
| Solid tumour | 50 (35.5) | 18 (40.0) | 15 (35.7) | 17 (31.5) | 0.69 |
| Haematologic tumour | 29 (20.6) | 7 (15.6) | 11 (26.2) | 11 (20.4) | |
| Non-oncologic disease | 62 (44.0) | 20 (44.4) | 16 (38.1) | 26 (48.1) | |
| Target population | |||||
| Adults only | 109 (77.3) | 28 (62.2) | 34 (81.0) | 47 (87.0) | 0.01 |
| Paediatric or mixed | 32 (22.7) | 17 (37.8) | 8 (19.0) | 7 (13.0) | |
| No. of GRADE tables, SoF [mean (SD)] | 3.5 (2.4) | 3.1 (1.6) | 3.8 (2.8) | 3.6 (2.7) | 0.72 |
| Total no. of studies in support | |||||
| 1 | 112 (79.4) | 37 (82.2) | 33 (78.6) | 42 (77.8) | 0.85 |
| > 1 | 29 (20.6) | 8 (17.8) | 9 (21.4) | 12 (22.2) | |
| No. of RCTs in support | |||||
| 0 | 25 (17.7) | 8 (17.8) | 7 (16.7) | 10 (18.5) | 0.63 |
| 1 | 95 (67.4) | 31 (68.9) | 31 (73.8) | 33 (61.1) | |
| > 1 | 21 (14.9) | 6 (13.3) | 4 (9.5) | 11 (20.4) | |
| No. of phase I/II clinical studies in support | |||||
| 0 | 110 (78.0) | 35 (77.8) | 32 (76.2) | 43 (79.6) | 0.92 |
| ≥ 1 | 31 (22.0) | 10 (22.2) | 10 (23.8) | 11 (20.4) | |
Data are expressed as n (%) unless otherwise specified
GRADE Grading of Recommendations, Assessment, Development, and Evaluation, RCTs randomized controlled trials, SD standard deviation, SoF summary of findings
aIn two cases, the drug report was focused on an agnostic drug
bAccording to the European Medicines Agency