FDA’s Drug Approvals

Charles Hooper and David Henderson are on the right track.

The Federal government requires pharmaceutical companies to prove that their drugs are both safe and effective before putting them on the market. Before 1962, companies needed to prove only safety. While there is some appeal to this two-hurdle approach, evidence suggests that there is only a slight benefit and a tremendous cost. With the COVID-19 pandemic sweeping the world, there has never been a better time to revoke the Food and Drug Administration’s efficacy requirement.

I suggest the FDA move to a two-stage approval process. The first stage should focus on safety: does the drug do no harm, at least compared with the condition it’s aimed at treating (because all drugs have side effects).  Once it’s determined the drug is safe, it should be released to the market, limited strictly to on-label use. Let prescribing doctors and patients determine whether the drug is useful, let market forces do their trick. The FDA’s imprimatur for this stage, should be limited to “safe as prescribed, not determined to be effective.”

The second stage should proceed without delay, overlapping the first stage to the extent feasible; in this stage, investigative focus should be on efficacy—does the drug actually have the effect on its target condition that’s intended. Only after the trials associated with this stage have been successfully completed could the drug receive its full-up FDA stamp of approval—and authorization for use, under prescription, off-label.

This modification to the pre-1962 requirement would cheapen development, and it would provide more drugs of greater utility faster to market and to the doctors and patients who use them.

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