TY  - JOUR
T1  - APproval of a tuberculosis drug based on a paradoxical surrogate measure
AU  - Avorn J
Y1  - 2013/04/03
N1  - 10.1001/jama.2013.623
JO  - JAMA
SP  - 1349
EP  - 1350
VL  - 309
IS  - 13
N2  - 
Ideally, drug approvals are based on large trials that randomize thousands of patients and measure actual clinical outcomes. By contrast, the pivotal studies for bedaquiline were relatively small; the initial finding on sputum culture conversion came from an 8-week trial of 47 patients. A follow-up study enrolled another 161 patients. By 24 weeks, 79% of patients taking bedaquiline in the second study had undergone sputum culture conversion, compared with 58% in the placebo group. The difference in each substudy was not significant at later points.1- 2

SN  - 0098-7484
M3  - doi: 10.1001/jama.2013.623
UR  - http://dx.doi.org/10.1001/jama.2013.623
ER  -