If even half of the account of the Food and Drug Administration review of the muscular dystrophy drug eteplirsen recounted by the editors of the Wall Street Journal is true, serious disciplinary action is required:
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. Janet Woodcock, head of the drug evaluation center, can overrule the department and the recommendations of an advisory panel.
The question is whether someone in the division is holding up an approval by Dr. Woodcock with what is known as a differing-professional-opinions proceeding, which is an agency process for handling scientific disputes. According to FDA manuals, this involves a panel review that can take weeks or more, and an appeals channel can escalate to FDA Commissioner Robert Califf. This is the only logical explanation for the continuing delay, as FDA could have rejected the drug already if the data were junk. (An FDA spokesman declined comment.)
The Federal Employees Liability Reform and Tort Compensation Act of 1988 provides an absolute shield to federal employees from civil suit whether they are doing their jobs properly or not, whether they are breaking the law or not, and whether they are acting within the discretion authorized in their official duties or not. We should return to the earlier, more limited common law standard under which officials were only shielded if they were carrying out their duties and acting within their authorized discretion. Agencies should not defend employees who are malfeasant, misfeasant, or nonfeasant.
We deserve public employees who are carrying out the people’s business in a fair, unbiased, and timely manner. That is even more urgent as the boundaries of government power expand.
I find the WSJ article suspect because it doesn’t report that the readouts of the continuing trial were ever actually given to the FDA, just that the company promised to provide them shortly. Did they provide them? WSJ doesn’t appear to know.
On the larger issue, exposing government employees to greater liability wouldn’t change anything. They would be exposed more for approving a drug which causes bad things to happen than they would for delaying approval of a drug that might help some people walk.
The problems of agency delay are usually addressed through deadlines on the agency, opportunity for judicial review, and proper calibration of the burden of proof. That is, the legislature creates the system which creates the incentives.
Not if they do their jobs within the guidelines and discretion they’re given.
Or, as Sam Clemens put it, the United States has no native criminal class. Excepting, of course, the Congress.
Almost all of these WSJ op-eds supporting a specific drug get in there because some special interest has influence with the WSJ board, or the board can use it to further its own ends. The drugs are usually bad, or totally cost-ineffective.
In this case, the company submitted a single study with 8 patients, and the results were negative. Rather than submit studies, they have families trying to pressure the FDA. Looks to me like the FDA is doing the right thing.
http://www.healthnewsreview.org/2016/06/whats-stake-fdas-decision-eteplirsen-experimental-muscular-dystrophy-drug/