FDA approves deadly drug … even after debating its ‘effectiveness’

Monday, October 10, 2016 by

It is often difficult to understand why government bureaucracies do some of the things they do, and obviously that includes the Food and Drug Administration, the agency responsible for approving some of the most dangerous drugs ever marketed.

In fact, the agency may just have done so again.

As reported by medical website STAT, the FDA’s chief, Dr. Robert Califf, wants a study about a Duchenne muscular dystrophy drug called eteplirsen retracted because he believes it was misleading. The problem is, the drug has already been approved.

Developed by Sarepta Therapeutics, the drug is the first to be approved for the rare genetic disease which leads to a steady erosion and wasting of muscles that eventually consigns boys to wheelchairs and an early grave. STAT reports that Duchenne was approved in spite of often rancorous disagreement out of the limelight among top FDA officials regarding the extent that the drug actually helps patients.

Central to the infighting was a 2013 study into whether the drug was able to produce levels of a protein called dystrophin in sufficient amounts. Without that protein, muscle fibers deteriorate and voluntary movement disappears.

The study, which of course was funded by the drug maker and co-authored by four of the pharmaceutical’s employees, found that the medicine produced enough dystrophin and allowed affected boys some ability to walk.

But Califf sided with a pair of FDA officials who also believe that the study, which was published in the journal Annals of Neurology, should be pulled.

“The publication, now known to be misleading, should probably be retracted by its authors,” Califf wrote in a memo, in which he touched on the internal dispute within his agency.

“In view of the scientific deficiencies identified in this analysis, I believe it would be appropriate to initiate a dialogue that would lead to a formal correction or retraction (as appropriate) of the published report,” the agency chief added in a footnote.

The disagreement was initiated in large part by various moves made by Dr. Janet Woodcock, the controversial manager of the FDA’s drug review division, who pushed hard for the approval of the Sarepta drug.

Though she reportedly had concerns about the study, she also argued with other agency officials who did not agree with her decision to approve the drug, as well the manner in which she advocated on behalf of the drug’s approval.

The trouble is, it’s unclear where the situation goes from here. Dr. Clifford Saper, editor of the Annals of Neurology, said that getting a paper retracted is no quick or easy thing.

“It takes more than a call by a politician for retraction of a paper. It takes actual evidence,” he told STAT. He added that if Califf has evidence that there are errors in the paper, he should produce it.

Meanwhile, another drug that is likely ineffective and perhaps even expensive will be permitted to remain on the market and in use.



FDA.gov [PDF]


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