Epstein: "A Taste of Government-Run Healthcare"
In his recent article in National Affairs, “Changing the FDA’s Culture,” Dr. Scott Gottlieb rightly takes the FDA to task for its retrograde attitudes on the approval of new drugs for sale in the United States market. But he is far too kind to the FDA. Its culture is beyond repair by external exhortation. The FDA is a bureaucratic monopolist. Currently, in the name of protecting the American public, the FDA represents a major systematic threat to drug innovation and public health. It should be stripped of its power to block the entry of new drugs and medical devices into general use.
The Case of Elaprase
In making his case for a more modest response, Gottlieb begins with a story of the drug Elaprase, which has been used since 2006 to treat Hunter’s Syndrome, a horrific but rare children’s disease. The disease is caused by the want of a single enzyme needed to break down sugar molecules inside the body. As sugar molecules build up in concentration, the disease cripples and often kills children from a very young age.
As Gottlieb notes, progress against the disease was slow, and the first signs of real hope appeared in the early 2000s when researchers found a way to develop enzyme replacements in related enzyme deficiency diseases. When Elaprase first became available in 2004, the sensible response for the FDA would have been to ensure that the product did not kill patients at small dosages, and then to let it into the marketplace, with due notification of its experimental nature and any known side effects.