This article originally appeared at Forbes and has been republished here with the author’s permission.
In the Trump administration, we can expect a new head of the FDA, one of the 4,000 or so political appointees. The post of FDA Commissioner is one of the most important in the government because the FDA regulates products worth more than $1 trillion, 25 cents of every consumer dollar. Those products, which encompass food, drugs, vaccines, medical devices and your dog’s flea medicine, affect every American in innumerable ways every day.
Moreover, the FDA is a “gatekeeper,” which means that it must issue affirmative approvals of many classes of products before they can be marketed.
And yet, appointments to this critical job have often seemed to be treated by the White House as something of an afterthought.
The current FDA commissioner, Dr. Robert Califf, assumed the position only in February 2016, so he has not really had time to make his mark. His predecessor, Dr. Margaret Hamburg, was there for seven years, however, and provides an exemplar of what we don’t want in a Trump administration incumbent.
Dr. Hamburg was intelligent and experienced in public health before her appointment to head the FDA, but she chose to be primarily a figurehead, traveling extensively and gushing constantly about her agency’s commitment to sound science and its superior performance, although often the record told a very different story.
Hamburg hewed to the wishes of her political masters even when they conflicted with science, federal law and common sense, and she made inexplicable errors of judgment, neglected to crack the whip when her minions dragged their feet on approving important medicines and other products, and failed to exercise appropriate scrutiny over “compounded drugs.” Examples of injurious delays include vaccines for Meningitis B, and pirfenidone, a drug to treat a fatal disease called idiopathic pulmonary fibrosis. The delays in approving these products, which had been widely approved abroad before FDA sanctioned them, resulted in a palpable and preventable body count in this country.
The FDA needs renewal. It is dysfunctional, suffering from cultural, organizational and management problems that have been exacerbated by White House micromanagement and congressional mandates and meddling. Too often, FDA officials have gotten away with performing like Herman Melville’s fictional character Bartleby, who responds repeatedly to requests from his employers with, “I would prefer not to.”
A particularly egregious example of nonfeasance on Hamburg’s watch was the agency’s unwillingness to confirm as GRAS (Generally Recognized As Safe) the proteins lysozyme and lactoferrin obtained from genetically engineered rice, a process called “biopharming.” Clinical research had shown that when the proteins, which occur naturally in human tears and breast milk, are added to oral rehydration solution to treat children with diarrhea, they both lessen the duration of symptoms and reduce the rate of recurrence.
The availability of such a fortified oral rehydration solution for people in the developing world would be a near-miraculous advance. By having withheld GRAS affirmation for lysozyme and lactoferrin–again, presumably for political reasons–Hamburg is responsible for untold numbers of preventable deaths in Africa, Asia and Latin America. She deserves no “profiles in courage” awards.
As a result of FDA’s policies and performacne, drug development by major U.S.-based drug companies is less than robust, with R&D costs way up and regulatory approvals flat; and there are worrisome shortages of many essential drugs. Some entire FDA-regulated sectors, such as “biopharming” and R&D on genetically engineered food animals, have virtually disappeared because of regulatory excesses or uncertainty.
Rebooting the FDA will be one of the toughest jobs in Washington, and the incumbent will need the exceptional qualities discussed below:
Superior management skills and experience. The FDA commissioner should be able to direct, manage and make the tough policy decisions for a $5 billion, 15,000-employee organization that is constantly in the news and under pressure from various stakeholders. Because the agency’s scope is so sweeping–encompassing drugs, vaccines, cardiac pacemakers, x-ray machines, condoms, home pregnancy-testing kits, artificial sweeteners, fat substitutes and tobacco, among other products, a single person cannot be expected to master the body of science, medicine, pharmacology, toxicology, engineering and law involved.
Therefore, the commissioner must have the skills to organize the agency efficiently and possess sufficient technical understanding to discern whether FDA’s professional staff are properly framing the issues and options. The incumbent will also need to address what many observers of FDA consider to be excessive risk-aversion. That will require what Dr. Janet Woodcock, the long-serving director of the Agency’s Center for Drug Evaluation and Research, has called a “cultural and cognitive transformation.”
Unassailable integrity and honesty. The commissioner’s public health decision-making must meld law, science and regulatory precedents. The incumbent needs to earn the respect of those who have a stake in the FDA’s policies and decisions–consumers, political leaders, industries and public interest groups. But in the end, science–not public opinion, politics (see below), pressure from special interests, or congressional grandstanding–must dictate policy and decision-making.
I was struck by the dissonance between a statement by Dr. Hamburg several years ago and the FDA’s performance where it counts—getting new medicines to patients. She bragged that “[p]reliminary results of reviews completed during FY 2010 indicate that FDA has the potential to meet or exceed almost all (11 of 12) FY 2010 review performance goals.” But 2010 was the worst year for drug approvals in a quarter century. This kind of disconnect is typical of federal bureaucrats: They create easily-met performance milestones that may have little correlation with the agency’s actual mission. As the old medical cliché goes, the operation was a success but the patient died.
Distanced from politics. Politics should be banished from the agency head’s role insofar as that is possible, with the commissioner taking the heat for unpopular decisions and telling truth to power. Many commissioners, including notably Hamburg and David Kessler, who headed the agency during the 1990’s, have deferred to their political bosses, not only on matters of policy (which is often appropriate), but also on decisions about individual products.
Some of Hamburg’s political capitulations were inexcusable. They include allowing the Secretary of HHS to overrule the FDA’s decision to let the Plan B morning-after pill be sold over the counter to young teenagers; and permitting the White House to hijack what should have been a routine, scientifically uncontroversial approval of a faster-growing, genetically engineered farmed salmon (but which was under review for more than 20 years). The FDA has also unnecessarily and inexplicably delayed approval of a single small field trial of mosquitoes genetically engineered to control disease-causing mosquitoes. (The same product has been extensively tested in other countries and is approved for commercial use in Brazil.)
It should be noted that these failures are endemic, from entry-level medical officers up the food chain to policymakers and the agency head. Will the new commissioner show more spine and integrity, making reforms a high priority? Time will tell.
Committed to regulatory reform. At a time when drug development should have been spurred by huge increases in R&D expenditures–which doubled to more than $51 billion between 2000 and 2013–and by the availability of numerous new, powerful technologies, drug approvals have been essentially flat for the past 15 years. Bringing a new drug to market now requires on average 10-15 years, and costs have skyrocketed to more than $2.5 billion—largely because FDA requirements have increased the length and number of clinical trials per marketing application and the number of procedures per patient. Another ominous statistic is that drug manufacturers recoup their R&D costs for only one in five approved drugs.
Under Hamburg, the FDA extended its authority beyond statutory limits. The Federal Food, Drug and Cosmetic Act requires, for example, that for a drug to be marketed, it must be shown to be safe and effective. But the agency has invented new, arbitrarily-applied criteria, including a demonstration of superiority compared to other medicines. Proving that a drug is better than existing drugs often is much more difficult and vastly more expensive than just proving that it is safe and effective, because if two medicines’ efficacy differs only marginally, the clinical trials must be very large in order to attain statistical significance. Moreover, even if two drugs are both found to be effective in 40% of patients, they may not be effective in the same 40%. If this new criterion were widely implemented, many new (odrugs useful for some patients would founder, reducing competition in the drug market, limiting doctors’ choices, and causing prices to rise.
The FDA needs to streamline its existing regulatory procedures and requirements, and the agency’s senior and mid-level managers must be made more accountable for their decisions, especially when they delay the availability of important new drugs, vaccines and medical devices to patients in need of them. And as former FDA Commissioner Frank Young used to admonish his minions, rules and regulations need to be tempered with common sense.
Distanced from politics. Politics should be banished from the agency head’s role insofar as that is possible, with the commissioner taking the heat for unpopular decisions and telling truth to power. Many commissioners, including notably Hamburg and David Kessler, who headed the agency during the 1990’s, have deferred to their political bosses, not only on matters of policy (which is often appropriate), but also on decisions about individual products.
Some of Hamburg’s political capitulations were inexcusable. They include allowing the Secretary of HHS to overrule the FDA’s decision to let the Plan B morning-after pill be sold over the counter to young teenagers; and permitting the White House to hijack what should have been a routine, scientifically uncontroversial approval of a faster-growing, genetically engineered farmed salmon (but which was under review for more than 20 years). The FDA has also unnecessarily and inexplicably delayed approval of a single small field trial of mosquitoes genetically engineered to control disease-causing mosquitoes. (The same product has been extensively tested in other countries and is approved for commercial use in Brazil.)
It should be noted that these failures are endemic, from entry-level medical officers up the food chain to policymakers and the agency head. Will the new commissioner show more spine and integrity, making reforms a high priority? Time will tell.
Committed to regulatory reform. At a time when drug development should have been spurred by huge increases in R&D expenditures–which doubled to more than $51 billion between 2000 and 2013–and by the availability of numerous new, powerful technologies, drug approvals have been essentially flat for the past 15 years. Bringing a new drug to market now requires on average 10-15 years, and costs have skyrocketed to more than $2.5 billion—largely because FDA requirements have increased the length and number of clinical trials per marketing application and the number of procedures per patient. Another ominous statistic is that drug manufacturers recoup their R&D costs for only one in five approved drugs.
Under Hamburg, the FDA extended its authority beyond statutory limits. The Federal Food, Drug and Cosmetic Act requires, for example, that for a drug to be marketed, it must be shown to be safe and effective. But the agency has invented new, arbitrarily-applied criteria, including a demonstration of superiority compared to other medicines. Proving that a drug is better than existing drugs often is much more difficult and vastly more expensive than just proving that it is safe and effective, because if two medicines’ efficacy differs only marginally, the clinical trials must be very large in order to attain statistical significance. Moreover, even if two drugs are both found to be effective in 40% of patients, they may not be effective in the same 40%. If this new criterion were widely implemented, many new (odrugs useful for some patients would founder, reducing competition in the drug market, limiting doctors’ choices, and causing prices to rise.
The FDA needs to streamline its existing regulatory procedures and requirements, and the agency’s senior and mid-level managers must be made more accountable for their decisions, especially when they delay the availability of important new drugs, vaccines and medical devices to patients in need of them. And as former FDA Commissioner Frank Young used to admonish his minions, rules and regulations need to be tempered with common sense.
During confirmation hearings, senators should scrutinize the nominee’s resume carefully and broach a broad spectrum of policy issues and product delays that affect public health. Whoever the nominee is, I wish him (or her) well.
Henry I. Miller, a physician, is the Robert Wesson Fellow in Scientific Philosophy & Public Policy at Stanford University’s Hoover Institution. He was the founding director of the FDA’s Office of Biotechnology. Follow him on Twitter @henryimiller.