Last month FDA granted permission for “expanded access” to an experimental medicine for Ebola virus infection. The drug, currently designated TKM-Ebola, has barely begun to be tested for safety and efficacy and is available in extremely limited quantities. It will be administered 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 set of priorities. To put it less charitably, FDA has been dragging its feet on the approval of critical products for the prevention or treatment of other fatal illnesses that are far more common in this country than Ebola – which has caused exactly zero deaths in Americans — so I was puzzled by regulators’ willingness to expend time and energy on a drug that will be used rarely, if at all, in the United States.
One example of a product that should be a high priority is Bexsero, a vaccine for meningitis B, or MenB, an infection which continues to plague U.S. college campuses. Over the last four decades, the threat from several types of bacterial meningitis has been significantly reduced by immunization. The first vaccine was approved in 1974, but a vaccine for MenB has taken much longer to develop. Bexsero was approved in 2013 by the European Union, Australia and Canada. It has not been approved in the United States, in spite of continuing outbreaks on college campuses, the most recent of which killed a Georgetown University student last month and a San Diego State University student on October 16.
Citizens have shown initiative and courage where bureaucrats have not. The mother of a student at the University of California, Santa Barbara, sent her son to England to be immunized with Bexsero. And the mother of a young woman who died in Michigan has organized bus trips for dozens of people, mostly college-age kids, from her home in Michigan to Windsor, Ontario, where she arranged for the group to be seen by a doctor, purchase the vaccine and have it administered. To draw attention to the urgent need for this vaccine, Rep. Erik Paulsen (R-MN), the co-chair of the Congressional Medical Technology Caucus, went on one of the trips and was vaccinated himself.
Another example is the sorry saga of a drug called pirfenidone, intended to treat a pulmonary disorder called idiopathic pulmonary fibrosis (IPF) which kills tens of thousands of Americans annually. The cause of the disease is unknown, and there were no FDA-approved therapies until on October 15 the FDA approved two drugs to treat it — finally. Pirfenidone had already been marketed in Europe (since 2011), Japan (2008), Canada (2012) and China. Pirfenidone was approved in the EU on the basis of three randomized, double-blind, placebo-controlled studies — one conducted in Japan and the other two in Europe and the United States.
So why not in this country until this month? In 2010, in spite of a recommendation for approval by an FDA advisory committee (comprised of outside experts), agency officials denied approval of the drug and demanded another major clinical study. The results, published in May of this year, were impressive. Pirfenidone markedly improved several laboratory and clinical measures of lung function and, most important of all, reduced the probability of death from IPF.
Between 2010 and the recent approval, more than 150,000 patients have died of IPF in the United States. Ironically, the FDA granted to pirfenidone “fast track, priority review, orphan product, and breakthrough designations.” And yet there were still unconscionable and unnecessary delays.
Economist Diana Furchtgott-Roth has described other similar delays in the approval of drugs to treat Duchenne muscular dystrophy, multiple sclerosis and diabetes.
The FDA’s priority-setting is distorted, seemingly influenced by factors other than data and the nation’s medical needs. The Ebola outbreak in Africa is on the front page, so FDA grants expanded access for an Ebola drug. And last spring, FDA 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, I don’t fault FDA on the merits, but 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. Facebook and Twitter? Should regulators set priorities and make decisions the way that players are chosen for baseball’s All-Star Game, with members of the public voting for their favorites?
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. But the civil servants at FDA have failed to hold up their end of the bargain; they bend like reeds in the shifting winds of current events. Americans deserve better.
Dr. Miller, a physician and molecular biologist, is the Robert Wesson Fellow in Scientific Philosophy and Public Policy at Stanford University’s Hoover Institution. He was the founding director of the FDA’s Office of Biotechnology.