From CMPI blogisto, Dr. Henry Miller …
In recent years, the costs of drug development have skyrocketed, with
direct and indirect expenses now exceeding $800 million to bring an average drug to market; and fewer than one in three drugs that are approved for marketing ever recoup their development costs. Even more ominous, the number of applications to FDA by industry for permission to market drugs has been steadily decreasing since 1995.
But drug and biotech companies spend more than $30 billion a year on
research, so how can that be? The reason is that regulators are
continually raising the bar for approval. For example, in just the past few years FDA officials have arbitrarily and unexpectedly directed clinical investigators to begin trials at inappropriately low dosages; limited approval of early stage studies only to single-dose, instead of dose-ranging, studies; demanded unnecessary, invasive procedures on patients; and even required that foreign trials be completed and the results submitted before the U.S. trials could begin. It’s bad enough that such an overly cautious mindset delays the availability of new drugs, but regulators’ increasing preoccupation with safety concerns may have become contagious: Drug manufacturers, too, seem to have begun to “err on the side of safety” to a degree that causes safe and effective drugs to be taken off the market voluntarily. An example is Tysabri, only the sixth medication approved — and the first in several years — for the treatment of Multiple Sclerosis (MS), a debilitating autoimmune disease that affects the central nervous system. The stunning results of the drug’s testing in clinical trials — which showed improvement in the quality of life and a reduction by more than half in the frequency of clinical relapses — induced FDA to grant accelerated approval last fall. MS patients eagerly put their names on waiting lists to get the medicine. But this ray of hope for MS sufferers was short-lived. By the time that several thousand patients were being treated with Tysabri, three deaths from a rare neurological disorder caused by a virus were reported. (Because the drug suppresses certain aspects of the immune response, regulators, clinicians and the drug’s developers had from the beginning been sensitive to the possibility of infections as a side effect.) Immediately — some would say prematurely — the manufacturers of the medicine voluntarily halted production and distribution, withdrew Tysabri from the market, and immediately initiated a full review of all patient data. MS victims and many neurologists were bitterly disappointed. Data obtained since the withdrawal have confirmed the drug’s efficacy and shown no additional drug-related deaths, so as a scientist, physician and
policy wonk, I wonder why Tysabri isn’t back on the market. Surely, this is a product that patients (with the approval of their physicians) should have the right to choose.