Table 1.
Variables detected on the appraisal document and innovativeness status (2017–2020)
| All diseases (n=77) | Oncology (n=49) | Non-oncology (n=28) | ||||||||||
| All medicines n (%) |
Innovative* n (%) |
Not innovative n (%) |
P value† | All medicines n (%) |
Innovative* n (%) |
Not innovative n (%) |
P value† | All medicines n (%) |
Innovative* n (%) |
Not innovative n (%) |
P value† | |
| CTS appraisal year | ||||||||||||
| 2017 | 28 (36.4) | 18 (64.3) | 10 (35.7) | 18 (36.7) | 12 (66.7) | 6 (33.3) | 10 (35.7) | 6 (60.0) | 4 (40.0) | |||
| 2018 | 25 (32.5) | 17 (68.0) | 8 (32.0) | 15 (30.6) | 10 (66.7) | 5 (33.3) | 10 (35.7) | 7 (70.0) | 3 (30.0) | |||
| 2019 | 24 (31.2) | 16 (66.7) | 8 (33.3) | 0.96 | 16 (32.7) | 13 (81.2) | 3 (18.8) | 0.57 | 8 (28.6) | 3 (37.5) | 5 (62.5) | 0.37 |
| Rare disease | ||||||||||||
| No | 34 (44.2) | 20 (58.8) | 14 (41.2) | 23 (46.9) | 17 (73.9) | 6 (26.1) | 11 (39.3) | 3 (27.3) | 8 (72.7) | |||
| Yes | 43 (55.8) | 31 (72.1) | 12 (27.9) | 0.22 | 26 (53.1) | 18 (69.2) | 8 (30.8) | 0.72 | 17 (60.7) | 13 (76.5) | 4 (23.5) | 0.02 |
| Disease | ||||||||||||
| Solid tumours | 30 (39.0) | 21 (70.0) | 9 (30.0) | 30 (61.2) | 21 (70.0) | 9 (30.0) | – | – | – | |||
| Haematological malignancies | 19 (24.7) | 14 (73.7) | 5 (26.3) | 19 (38.8) | 14 (73.7) | 5 (26.3) | 0.78 | – | – | – | ||
| Infectious diseases | 5 (6.5) | 3 (60.0) | 2 (40.0) | – | – | – | 5 (17.9) | 3 (60.0) | 2 (40.0) | |||
| Autoimmune diseases | 3 (3.9) | 1 (33.3) | 2 (66.7) | – | – | – | 3 (10.7) | 1 (33.3) | 2 (66.7) | |||
| Other | 20 (26.0) | 12 (60.0) | 8 (40.0) | 0.64 | – | – | – | 20 (71.4) | 12 (60.0) | 8 (40.0) | 0.68 | |
| Population | ||||||||||||
| Adults only | 65 (84.4) | 42 (64.6) | 23 (35.4) | 46 (93.9) | 33 (71.7) | 13 (28.3) | 19 (67.9) | 9 (47.4) | 10 (52.6) | |||
| Paediatric or mixed | 12 (15.6) | 9 (75.0) | 3 (25.0) | 0.74 | 3 (6.1) | 2 (66.7) | 1 (33.3) | 0.99 | 9 (32.1) | 7 (77.8) | 2 (22.2) | 0.22 |
| Mean number SoF (SD) | 3.4 (2.9) | 3.1 (2.6) | 3.8 (3.4) | 0.34 | 2.5 (2.2) | 2.5 (2.2) | 2.4 (2.1) | 0.69 | 4.9 (3.4) | 4.4 (2.8) | 5.5 (4.0) | 0.42 |
| Clinical Studies (n) | ||||||||||||
| 1 | 61 (79.2) | 41 (67.2) | 20 (32.8) | 45 (91.8) | 31 (68.9) | 14 (31.1) | 16 (57.1) | 10 (62.5) | 6 (37.5) | |||
| >1 | 16 (20.8) | 10 (62.5) | 6 (37.5) | 0.72 | 4 (8.2) | 4 (100.0) | 0 (0.0) | 0.31 | 12 (42.9) | 6 (50.0) | 6 (50.0) | 0.51 |
| RCT (n) | ||||||||||||
| 0 | 15 (19.5) | 10 (66.7) | 5 (33.3) | 12 (24.5) | 7 (58.3) | 5 (41.7) | 3 (10.7) | 3 (100.0) | 0 (0.0) | |||
| 1 | 49 (63.6) | 34 (69.4) | 15 (30.6) | 34 (69.4) | 25 (73.5) | 9 (26.5) | 15 (53.6) | 9 (60.0) | 6 (40.0) | |||
| >1 | 13 (16.9) | 7 (53.8) | 6 (46.1) | 0.57 | 3 (6.1) | 3 (100.0) | 0 (0.0) | 0.32 | 10 (35.7) | 4 (40.0) | 6 (60.0) | 0.17 |
| Clinical trials phase I/II (n) | ||||||||||||
| 0 | 59 (76.6) | 38 (64.4) | 21 (35.6) | 37 (75.5) | 28 (75.7) | 9 (24.3) | 22 (78.6) | 10 (45.4) | 12 (54.6) | |||
| ≥1 | 18 (23.4) | 13 (72.2) | 5 (27.8) | 0.54 | 12 (24.5) | 7 (58.3) | 5 (41.7) | 0.29 | 6 (21.4) | 6 (100.0) | 0 (0.0) | 0.02 |
*Innovative status includes fully and conditionally innovative.
†Comparisons between innovative and non-innovative outcomes were performed using a contingency table with the χ2 or Fisher’s exact test, as appropriate for categorical data. Continuous data were analysed using a Student’s t-test, after checking for normal distribution (based on the Shapiro-Wilk statistic), or a Wilcoxon rank-sum test otherwise.
CTS, Technical-Scientific Committee; RCT, randomised clinical trial; SoF, Summaries of Findings.