Table 3.
Characteristics of main studies supporting marketing authorisation of cancer medicines in the US and in Brazil.
| Main clinical trial characteristics | US/FDA |
Brazil/Anvisa |
p-value |
|---|---|---|---|
| n (%) | n (%) | ||
| Drugs with Randomised Controlled Trial (RCT) as main studies | 34/56 (60.7) | 42/56 (75.0) | 0.16 |
| Primary endpointa | |||
| Overall survival (OS) | 10/56 (17.9) | 16/56 (28.6) | 0.27 |
| Progression free-survival (PFS) | 17/56 (30.4) | 23/56 (41.1) | 0.34 |
| Other | 3256 (57.1) | 23/56 (41.1) | 0.016 |
| Type of control arm of the RCTs | |||
| Active comparator | 12/56 (21.4) | 24/56 (42.9) | 0.025 |
| Placebo controlled | 16/56 (28.6) | 19/56 (33.9) | 0.064 |
| Add-on therapy | 6/56 (10.7) | 6/56 (10.7) | 1 |
| Adequacy of control arm | |||
| Optimal | 13/56 (23.2) | 19/56 (33.9) | 0.30 |
| Suboptimal | 9/56 (16.1) | 13/56 (23.2) | 0.48 |
| Clinically relevant outcome | |||
| Overall Survival Benefit | 12/56 (21.4) | 24/56 (42.9) | 0.025 |
| Substantial Clinical Benefitb | 11/56 (19.6) | 16/56 (28.6) | 0.42 |
Bold p-value indicates that the correspondent difference is statistically significant.
a
Some studies had more than one primary endpoint.
b
European Society of Medical Oncology—Magnitude of Clinical Benefit Scale (ESMO-MCBS) ≥4, data available with ESMO-MCBS classification n = 26 drugs at the FDA and n = 29 drugs at Anvisa.