Monday, December 19, 1994
Unlike Charles Barkley, that other well-known Republican, Newt Gingrich, would love to be one of our role models. We think the jury is going to remain out on that score. Nevertheless, two decisions last week by the FDA's Oncologic Drugs Advisory Committee - recommendations against approval of U.S. Bioscience Inc.'s Ethyol chemoprotectant and Rhone-Poulenc Rorer's Taxotere chemotherapeutic - have raised some decidedly Newt-like thoughts in these parts.
In this era of widespread regulatory overkill, and a growing sense among the public that government has become too intrusive, the bureaucratic premises underlying the two recommendations simply beg challenge.
When it comes to life-threatening conditions, and for treatments for the terminally ill, the bar for approval has been raised too high and the regulators are too quick to substitute their judgement for that of the front-line clinicians who ultimately must treat the patients.
This situation has a cost to companies in terms of drug development expenses and, far more importantly, to patients in terms of delayed access to drugs and of drugs that may never get approved or developed at all.
Our rationale has to do both with the fact that clinical trial conditions often pose more rigorous challenges for drugs than they would face in real use settings, and with our preference to let the market - the physicians and patients - rather than the bureaucracy make decisions in cases where safe products may be marginally effective, and in cases where there are tradeoffs between efficacy and toxicity.
This is a tricky road to tread, because so-called public interest groups can dispense anecdotal horror stories about under-regulation, while the costs of over-regulation are more diffuse and harder to quantify. Witness the publicity surrounding the deaths of hepatitis patients taking FIAU in clinical trials. By comparison, we doubt there will be one article in the general media that attempts to estimate the number of patients who might have had their lives lengthened by using Taxotere. We've certainly never seen such an estimate for sepsis drugs, where the bar to proving efficacy is impossibly high.
But the fact that AIDS patients, and to a lesser extent Alzheimer's patients, have been able to win concessions in this area, is proof that it's possible to change the weight of evidence required for regulatory approval where the political will to do so exists.
Let the market decide
Oncology is a good example of the reality of market-based decision making, because the proper usage of cancer drugs is routinely established after approval, not before, through experimentation and off-label use.