In the latest Milken Institute Review, Nobel laureaute Gary Becker argues (sign up required) that the FDA should permit drugs to be sold once they have passed a safety standard, i.e. a return to the pre-1962 system. He writes:
…a return to a safety standard alone would lower costs and raise the number of therapeutic compounds available. In particular, this would include more drugs from small biotech firms that do not have the deep pockets to invest in extended efficacy trials. And the resulting increase in competition would mean lower prices – without the bureaucratic burden of price controls…
Elimination of the efficacy requirement would give patients, rather than the FDA, the ultimate responsibility of deciding which drugs to try…To be sure, some sick individuals would try ineffective treatments that would otherwise have been prevented from reaching market under present FDA regulations. But the quantity of reliable health information now available with only a little initiative is many times greater than when the efficacy standard was introduced four decades ago.
Dan Klein and I have written extensively on this issue at our web site, FDAReview.org, and in our latest paper Do Off Label Drug Practices Argue Against FDA Efficacy Requirements?