TY  - JOUR
T1  - MEga-trials for blockbusters
AU  - Ioannidis JA
Y1  - 2013/01/16
N1  - 10.1001/jama.2012.168095
JO  - JAMA
SP  - 239
EP  - 240
VL  - 309
IS  - 3
N2  - 
The conduct of very large, simple trials (mega-trials) is uncommon and faces several challenges, in particular cost and difficulty in patient recruitment. However, these challenges can be overcome when interventions are widely used by hundreds of thousands of individuals and when there is a potential profit to accommodate the trial cost. Accordingly, every licensed intervention with annual sales that exceed $1 billion, ie, a blockbuster, should have at least 1 trial performed with at least 10 000 patients randomized to the intervention of interest and as many randomized either to placebo (if deemed to be a reasonable choice) or to another active intervention that is the least expensive effective intervention available. The comparison drug can be a generic drug with a well-established effectiveness and safety profile.

SN  - 0098-7484
M3  - doi: 10.1001/jama.2012.168095
UR  - http://dx.doi.org/10.1001/jama.2012.168095
ER  -