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
 201728 (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)
 201825 (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)
 201924 (31.2)16 (66.7)8 (33.3)0.9616 (32.7)13 (81.2)3 (18.8)0.578 (28.6)3 (37.5)5 (62.5)0.37
Rare disease
 No34 (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)
 Yes43 (55.8)31 (72.1)12 (27.9)0.2226 (53.1)18 (69.2)8 (30.8)0.7217 (60.7)13 (76.5)4 (23.5)0.02
Disease
 Solid tumours30 (39.0)21 (70.0)9 (30.0)30 (61.2)21 (70.0)9 (30.0)
 Haematological malignancies19 (24.7)14 (73.7)5 (26.3)19 (38.8)14 (73.7)5 (26.3)0.78
 Infectious diseases5 (6.5)3 (60.0)2 (40.0)5 (17.9)3 (60.0)2 (40.0)
 Autoimmune diseases3 (3.9)1 (33.3)2 (66.7)3 (10.7)1 (33.3)2 (66.7)
 Other20 (26.0)12 (60.0)8 (40.0)0.6420 (71.4)12 (60.0)8 (40.0)0.68
Population
 Adults only65 (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 mixed12 (15.6)9 (75.0)3 (25.0)0.743 (6.1)2 (66.7)1 (33.3)0.999 (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)0.342.5 (2.2)2.5 (2.2)2.4 (2.1)0.694.9 (3.4)4.4 (2.8)5.5 (4.0)0.42
Clinical Studies (n)
 161 (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)
 >116 (20.8)10 (62.5)6 (37.5)0.724 (8.2)4 (100.0)0 (0.0)0.3112 (42.9)6 (50.0)6 (50.0)0.51
RCT (n)
 015 (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)
 149 (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)
 >113 (16.9)7 (53.8)6 (46.1)0.573 (6.1)3 (100.0)0 (0.0)0.3210 (35.7)4 (40.0)6 (60.0)0.17
Clinical trials phase I/II (n)
 059 (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)
 ≥118 (23.4)13 (72.2)5 (27.8)0.5412 (24.5)7 (58.3)5 (41.7)0.296 (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.