In his letter to The Wall Street Journal‘s Letters section, Todd Lorenz, a Stanford University employee degreed as a Medical Doctor, pushed for FDA to continue to pass on the efficacy of a new drug as a condition for approving it, never minding that that would drastically slow approval and subsequent availability.
There is no way to know for certain if drugs work without doing efficacy studies in humans. Preclinical and animal studies, while helpful, can’t predict with confidence which drugs will be useful. Most investigational cancer drugs that go into the clinic have been shown to work in animal models. Most don’t work in patients.
In a truly competitive free market, those that don’t work won’t stay in the market for long. Delaying approval until efficacy can be “proven,” though, denies cancer patients access to those drugs that do work, unnecessarily—unconscionably—risking their lives. Lorenz closed with this:
The answer, then, is to approve drugs after they’ve been demonstrated to be safe. Yet no drug is completely safe; some can lead to substantial adverse reactions. It may be acceptable to prescribe drugs with such profiles if the diseases they are intended to treat are serious enough to warrant the risk. The choice to use any drug in a particular patient always depends on such a cost-benefit analysis. Without an objective assessment of efficacy, no such determination is possible.
No drug is ever completely efficacious, either. Even so, Lorenz contradicted his call for a cost-benefit analysis with that repeated demand for an objective assessment of efficacy. He ignores the simple fact that that cost-benefit analysis is best done—is most effectively done—by the patient and his doctor, not by Government. The benefits and costs of a particular drug treatment can only be assessed empirically by those two critical analyzers acting in a medical drug market that is competitively fed by safe drugs. Those empirically collected use and outcome data will determine efficacy, and they will do so far faster and far more thoroughly than can a government agency populated by bureaucrats who happen to have medical degrees of one sort or another, and who hold out for repeated trials with sample sizes that are miniscule relative to the target population, even if those sample sizes argued to be statistically significant.
UCLA Emeritus Professor James Meyer, also the proud possessor of a degree as a Medical Doctor, complemented Lorenz with his own non sequitur.
Messrs Hooper and Steiner [Deregulation Can Make Medications Cheaper] argue that the cost of new drugs could be greatly reduced if the FDA focused only on their safety. Maybe so. But this overlooks that the federal government has had a major and increasing interest in efficacy since the passage of Medicare (1965), the growing responsibility for veteran care since the Vietnam War (1965) and the passage of ObamaCare (2010).
You bet it does. Those agencies have burgeoning populations of bureaucrats to keep employed and to keep expanding. Never mind that bit about denying access to safe drugs by those who need them until a collection of bureaucrats gets around to approving “efficacy.”
