Philadelphia Reflections

A New Food and Drug Category

Nervous System

An editorial in the April 7, 2006 Wall Street Journal rouses a proposal germinating in my mind for twenty years, a new compassionate status of pharmaceutical regulation for dangerous but effective new therapeutic agents, not presently provided for in law.

The editorial concerned a new drug treatment for multiple sclerosis, a crippling and usually ultimately fatal condition which destroys the nervous system. In a hearing of the tests of this new drug, we hear numbers like 85% total remission. Unfortunately, we also hear of four patients who died, quite possibly from the treatment. From that point on, you could write the reports from memory. Patients come forward, declaring their willingness to run any risk to get the drug; politicians are sympathetic. The Food and Drug Administration feels forced to withhold the drug from the market until the situation is clarified; other politicians are sympathetic. We are reminded of thalidomide, which was luckily withheld from the American market until the German experience showed that pregnant women who took thalidomide could have babies without arms or legs. This lucky illustration of the value of bureaucratic procrastination gave Senator Kefauver the strength to push through an amendment bearing his name, demanding proof of efficacy, something quite irrelevant to the safety of thalidomide. Seldom mentioned is the fact that thalidomide has since proved to be quite useful for the bone cancer called Multiple Myeloma; this value took a very long time to surface, under the circumstances. The efficacy rule accomplished the purpose, however, by delaying all drug approvals, unfortunately also shortening the time left for patent coverage, and vastly increasing the cost of drugs by shortening the time on a patent to recover development costs. After thirty years of hearing the same arias re-echoing about one drug introduction after another, it begins to look unlikely that Congress will ever be able to stand the political heat which would be generated by a serious attempt to fix this problem. But surely that can't be allowed to continue forever.

Taking the multiple sclerosis treatment as an example, the patients want the drug, and their doctors want to use the drug but they don't want to be sued or prosecuted. It's almost certain that most of these patients would sign anything, accept any risk, even agree to a shortened life expectancy in return for an 85% chance of stopping the disease in its tracks. All the doctors want is to be held harmless by the courts for exercising reasonable care under the circumstances. The same is probably true in a political sense, for the FDA and Congress, who surely would be satisfied to have the doctors take the blame for any problems encountered. The drug companies want to preserve their patent protection, surely that can be accommodated. Perhaps they should be asked to contribute to a fund for the victims of unforeseen accidents; they would probably agree. What would satisfy the trial bar is less certain; they should be asked to stipulate something. As a matter of fact, anyone at all who has qualms about such a system of special defined exemptions should be asked to come forward with serious suggestions. There would then ensue a lengthy and ultimately tedious debate about details.

This would all take some time; at the end of it, someone would have to make some arbitrary decisions. But at least we could expect more progress than we have seen in the last thirty years of indecision.

Originally published: Thursday, June 22, 2006; most-recently modified: Wednesday, May 22, 2019