The Food and Drug Administration just granted permission for “expanded access” to an experimental medicine for Ebola. It’s OK as far as it goes, but it’s an exception to the FDA’s reluctance to approve the use of life-saving products.
Safety and efficacy testing of the drug, designated TKM-Ebola, has barely begun, and quantities are extremely limited. It will be given to patients with confirmed or suspected Ebola infections, but not as part of a clinical trial.
As a 15-year veteran of the FDA, that decision surprised me — not because it was bad on the merits, but because it represents a peculiar setting of priorities.
To put it less charitably, FDA routinely drags its feet on the approval of products critical for the prevention or treatment of fatal diseases that are far more common in this country than Ebola.
Why expend the agency’s time and energy on a drug that will be used rarely, if at all, in the United States?
Consider the saga of Bexsero, a vaccine for meningitis B.
Since the first vaccine for bacterial meningitis was approved in 1974, immunization has significantly reduced the threat — but a vaccine for MenB took much longer to develop.
Bexsero was approved last year by the European Union, Australia and Canada — but it still hasn’t gotten an FDA OK, though outbreaks continue to occur on US campuses. (The most recent one killed a Georgetown University student last month.)
Citizens have shown initiative while bureaucrats stall. The mother of a UC-Santa Barbara student sent her son to England to be immunized with Bexsero.
And the mother of a woman who died in Michigan has organized bus trips for dozens of people , mostly college-age kids, to Windsor, Ontario, where she arranged for the group to be seen by a doctor and vaccinated.
Another example: pirfenidone, a drug meant to treat a pulmonary disorder called idiopathic pulmonary fibrosis , or IPF, which kills tens of thousands of Americans each year. The disease’s cause is unknown, and there are no FDA-approved therapies.
But pirfenidone is marketed in Europe (since 2011), Japan (2008), Canada (2012) and China. The drug won EU approval on the basis of three randomized, double-blind, placebo-controlled studies — one done in Japan and the other two in Europe and the United States.
Why is the FDA waiting? In 2010, an advisory committee of outside experts recommended approval, but agency officials demanded another major clinical study.
The results, published last May, were impressive. Pirfenidone markedly improved several laboratory and clinical measures of lung function and, most important, reduced the probability of death from IPF.
The FDA will probably OK the drug by year’s end. But in four years of delay, IPF will have killed more than 150,000 US patients.
Economist Diana Furchtgott-Roth has described similar delays in the approval of drugs to treat Duchenne muscular dystrophy, multiple sclerosis and diabetes.
It seems (literally) dead obvious: The FDA bases priorities on factors other than data and the nation’s medical needs. Africa’s Ebola outbreak is front-page news, so the FDA grants expanded access for an Ebola drug.
Similarly, the FDA last spring brokered the creation of a clinical trial specifically to get an experimental anti-viral drug to a young cancer patient suffering from an adenovirus infection after a bone marrow transplant.
Again, fine on the merits — except that this effort was expended for a single patient and only after the agency was subjected to “intense pressure” from the public on Facebook and Twitter.
This sort of decision-making violates the social contract between civil servants and the public:
In return for lifetime tenure, bureaucrats are supposed to make decisions that are dispassionate, data-driven and in the public interest.
Instead, the FDA moves faster on life-saving treatments mainly when it knows the public is watching.
FDA civil servants have failed miserably to hold up their end of the bargain. We deserve better.
Source: New York Post