ABSTRACT
Should conventional randomized clinical trials provide the standard of safety and efficacy when testing targeted treatments for cancer? Should we make amendments to our current regulatory standard, stick to it, or dispense with it? I am going to maintain that, under certain circumstances, smaller phase II trials provide good enough grounds to grant regulatory approval for targeted therapies. My argument will hinge on the size of trial population, showing how this size is important not only for scientific considerations, but also for ethical and political reasons. The current system was designed to provide massive consumer protection at a point when our understanding of the biology of cancer was still relatively poor and statistical tests gave the only solid evidence about treatment effects. With targeted therapies, risks are hedged in a way that allows patients (if well informed) to make decisions for themselves, instead of deferring on pharmaceutical regulators.
