Category | Variable | First indication n (%) | Subsequent indication n (%) | P value |
---|---|---|---|---|
REGULATORY APPROVAL | ||||
Type of MA granted (all agencies) | Standard | 61 (51%) | 207 (74%) | 0.001 |
Conditional | 34 (29%) | 39 (14%) | ||
Priority review | 24 (20%) | 33 (12%) | ||
Type of MA granted (excluding EMA) | Standard | 47 (53%) | 146 (69%) | |
Conditional | 24 (27%) | 36 (16%) | 0.032 | |
Priority review | 18 (20%) | 31 (15%) | ||
Orphan Designationa | Yes | 55 (46%) | 65 (23%) | < 0.0001 |
No | 64 (54%) | 214 (77%) | ||
CLINICAL EVIDENCE | ||||
Pivotal trial design | Phase II single arm | 42 (35%) | 56 (20%) | 0.009 |
Phase III placebo RCT | 30 (25%) | 76 (27%) | ||
Phase III head-to-head | 39 (33%) | 129 (46%) | ||
Other | 8 (7%) | 18 (6%) | ||
Type of primary endpoint | Clinical | 28 (24%) | 49 (18%) | |
Surrogate | 81 (68%) | 194 (69%) | 0.221 | |
Co-primary | 10 (8%) | 36 (13%) | ||
Trial size | Number of enrolled patients Mean [ 95% CI] | 486 [421 – 550] | 555 [504 -605] | 0.125 |
MCBSb | Score of 1 | 54 (48%) | 88 (35%) | 0.012 |
Score of 2 or 3 | 22 (20%) | 85 (34%) | ||
Score of 4 or 5 | 35 (32%) | 80 (31%) | ||
HTA OUTCOMESc | ||||
G-BA | Proof of added benefit | 25 (86%) | 26 (45%) | 0.004 |
Lesser/no added benefit | 4 (14%) | 32 (57%) | ||
HAS | Reimbursed | 27 (96%) | 54 (90%) | 0.299 |
Not-reimbursed | 1 (4%) | 6 (10%) | ||
NICE | List/List with Criteria | 26 (96%) | 43 (90%) | 0.304 |
Do not list | 1 (4%) | 5 (10%) | ||
SMC | List/List with Criteria | 23 (88%) | 43 (86%) | 0.763 |
Do not list | 3 (12%) | 7 (14%) | ||
CADTH | List/List with Criteria | 22 (96%) | 41 (84%) | 0.152 |
Do not list | 1 (4%) | 8 (16%) | ||
PBAC | List/List with Criteria | 23 (92%) | 33 (66%) | 0.015 |
Do not list | 2 (8%) | 17 (34%) |