In their recent article on NRO, Bob Goldberg and Peter Pitts took issue with a previous article published on NRO written by me, criticizing the FDA. The gist of their criticism was that my article was an ill-informed, unfair, and misdirected rant. They were wrong, and their apologies for the FDA only contribute to the agency’s problems.
The authors accuse me of being fixated with my “one and only major FDA reform idea: turning the FDA into a body that certifies the ability of nongovernmental organizations to approve drugs.” Anyone remotely familiar with my research on the FDA would know that this is false. It is true that such an approach works well for medical device regulation in Europe and in the United States for more than 20,000 categories of products certified by Nationally Recognized Testing Laboratories–the prototype of which is Underwriters Laboratories–but I have proposed a variety of mechanisms, major and minor, to make the FDA more efficient, accountable, and responsive to the needs of patients. For example, in an op-ed, “The Curse of Too Much Caution,” published in the Wall Street Journal in November 2004, I wrote:
If we are to balance drug safety, innovation in R&D, and the availability and price of new medicines, we must find a way to make regulators accountable for costly errors of all kinds. One way would be to create a vigorous, independent agency ombudsman that could compel regulators to act in the public interest. The office would have to possess the following attributes: (1) independence from the agency and the FDA commissioner; (2) access to independent expertise in relevant disciplines, including medicine, pharmacology, science, regulation, and law; and (3) the power to levy sanctions against FDA employees found to be responsible, individually or collectively, for flawed decisions or policies that constitute severe, avoidable errors.
I also wrote:
After the agency approves a new drug for marketing, physicians may prescribe it for indications other than the specific ones for which the FDA has granted marketing approval (and which are included in the labeling). This “off-label” use, which is extremely common in specialties such as oncology, pediatrics, obstetrics, and infectious disease, is vitally important as a source of medical innovation and to bring the benefit of new medical knowledge rapidly to patients. It allows physicians to take advantage of the most current research and experience concerning a drug’s properties for the benefit of their patients. But the FDA has tenaciously restricted and interfered with companies’ ability to make information about these off-label uses available — even if it has been peer-reviewed and is contained in journal articles and textbooks. . . FDA should . . . develop a means to offer doctors and patients current information on successful off-label uses of approved drugs.
The passage above appeared on Peter Pitts’s own website.
I am not some dyspeptic, academic, armchair regulator who likes to pontificate but lacks hands-on experience with regulation. I was an FDA official–a civil servant–for 15 years. In 1982, the group I headed approved human insulin, the first biopharmaceutical, in just five months, at a time when the average was 31.5 months. (And it was not easy getting the risk-averse bureaucracy to concur, I assure you.) A decade later, when I was the director of the FDA’s Office of Biotechnology, the agency published what remains the only genuinely risk-based and scientifically defensible policy toward biotechnology-derived foods anywhere in the world.
I take no joy in calling attention to the deterioration in FDA’s management and performance during the past decade, or to the current regulatory obstacles to the development of new drugs. I did not make up the facts I cited in my article showing the FDA’s increasing inability to approve drugs in an efficient manner. And all of this has come in spite of drug developers’ spending more than $50 billion a year on R&D, and in spite of the fact that “biotechnology, combinatorial chemistry, sequencing of the human genome, improved purification techniques, and other innovations have made drug research more efficient,” as I have written, as quoted by Goldberg and Pitts.
I am not alone in calling attention to the endemic risk-aversion that plagues the FDA. As I related in my article:
As former FDA General Counsel Peter Barton Hutt has observed, “FDA employees have been praised only for refusing to approve a new drug, not for making a courageous judgment to approve a new drug that has in fact helped patients and advanced the public health.” His views are echoed by former FDA Commissioner Alexander Schmidt: ”In all our FDA history, we are unable to find a single instance where a Congressional committee investigated the failure of FDA to approve a new drug. But the times when hearings have been held to criticize our approval of a new drug have been so frequent that we have not been able to count them. The message to FDA staff could not be clearer.”
And that clear message has been heeded: FDA officials now are defensive, almost to the point of paralysis.
The FDA’s Critical Path Initiative to stimulate drug development has many worthwhile elements, but it is too little, too late, and hardly a substitute for the introduction of new mechanisms to redress the agency’s pathological risk-aversion. As I wrote in “To America’s Health: A Proposal to Reform the FDA” (Hoover Institution Press, 2000):
Lifetime tenure for civil servants–which itself exacts costs–is supposed to free regulators to consider only the public interest as they render decisions. Instead, it has given us the worst of both worlds: On the one hand, it has become virtually impossible to remove incompetent or adversarial federal civil servants; on the other, we are forced to live with decision-making by federal officials that is frequently influenced by perceived risks to their careers.
That’s the kind of fundamental problem confronted by the FDA–and by an aging American population increasingly in need of new and innovative therapies. It’s a problem that the FDA is not taking appropriate actions to solve.
I am proud of my government service, my continuing commitment to science-based public policy, and my numerous published articles and books. However, there is one area of scholarship related to FDA regulation in which I admit to abject failure. As a member of the advisory board of Bob Goldberg’s and Peter Pitts’s Center for Medicine in the Public Interest, I have been unable to educate and enlighten them. And because they have become apologists for the FDA’s failures, I am no longer willing to lend credibility to their organization by remaining on its advisory board. It must fall to others to help them to understand that the health of Americans depends more on disciplined, science-driven public policy than on undeserved paeans to cronies in government.
—Henry I. Miller, a physician and fellow at the Hoover Institution, headed the FDA’s Office of Biotechnology from 1989 to 1993. Barron’s selected his most recent book, The Frankenfood Myth, one of the 25 Best Books of 2004.