Here are excerpts from a recent editorial titled
"Heart of Bureaucratic Darkness - Why won’t the FDA make a decision on a muscular dystrophy drug?":
"The Food and Drug Administration is months late deciding whether to approve a drug for muscular dystrophy, and by now there’s only one explanation: Some of the agency’s staff have exploited FDA processes to evade accountability for an unprofessional review.
Ten of 12 boys with Duchenne muscular dystrophy walk after four years of treatment on eteplirsen, which produces the protein dystrophin. FDA has pushed off approval for the first-in-class treatment, asking for more time or even months-long delays blamed on snow. In June FDA asked the drug’s sponsor, Sarepta Therapeutics, for readouts from a continuing trial, which the company said it would provide shortly. Since then, radio silence from the FDA.
It’s no secret that the drug’s detractors are career employees in the neurology division, two of whom opposed approval at a public FDA meeting in April: Eric Bastings, deputy director of the division; and Ronald Farkas, a clinical team leader. The pair say the drug doesn’t produce “enough” dystrophin, or maybe any dystrophin, depending on which day you ask....
Drs. Farkas and Bastings have lost the public’s confidence that they can conduct a fair review, and no bureaucratic proceeding should stop the FDA from reviewing their fitness for service. A cleaning out is overdue: The neurology division takes 600 days on average to approve a drug, according to a Manhattan Institute analysis. That’s three times the figure for the oncology division, which is hardly known for promptness, and neurology’s workload is below average for the agency.
FDA could issue a decision in a day or in a month, and no one knows. Meanwhile, parents with Duchenne children are swallowing decisions like: Should I enroll my son in a risky clinical trial when a drug that works might be approved any day now? What’s hideous is that parents are forced to make choices based on the little they can glean about a dysfunctional bureaucracy."
The full article is here: