The FDA needs reform. Biden’s nominee is not the person to do it
By Henry I. Miller, M.S., M.D. and Jeff Stier
When President Joe Biden nominated Obama-era Food and Drug Administration Commissioner Dr. Robert Califf to return to his old post, he made what is widely seen as a safe, if uninspired, choice.
Califf is a distinguished cardiologist and clinical trial specialist, but the day-to-day regulatory decision-making happens at the organizational levels below the commissioner. The FDA, a huge and critically important but dysfunctional organization, now needs a bold, clear-thinking reformer, but Califf, the ultimate insider, has the ideal resume to keep it on the wrong path.
Government regulation offers some reassurance to the public, to be sure, but when it is wrongheaded or merely fails to be cost-effective, it actually costs lives: directly by withholding life-saving and life-enhancing products and indirectly by diverting societal resources to gratuitous regulatory compliance.
The stakes are high. The FDA’s purview is wide, regulating pharmaceuticals, medical devices, food, and tobacco products that account for about 20 cents of every consumer dollar. The FDA has pushed the average cost (including out-of-pocket expenses and opportunity costs) to bring a new drug to market to over $2.5 billion . That ensures that many new drugs will have a hefty price tag and that others won’t get developed at all.
Stat News’s glowing coverage of the nomination makes our point: “Califf might not get the credit, but he was actually one of the FDA officials at the center of the agency’s early efforts to regulate e-cigarette companies.” Califf’s approach has led to awful outcomes. The FDA has almost entirely extinguished the legal market for lower-risk alternatives used by adults to quit smoking. At the same time, unscrupulous businesses continue to provide headline-grabbing talking points to opponents of science-based tobacco harm reduction. And now, it looks as though the Supreme Court may need to decide whether the FDA’s messy rulemaking ran afoul of administrative law .
Putting the FDA on the right track will require toughness and discipline at a federal agency where more than 99.9% of the employees are civil servants who cannot be fired, even for incompetence or insubordination. Therefore, the most important role of the commissioner is to ensure that policies are scientifically defensible, that the agency’s culture reflects the appropriate level of risk aversion, and that managers manage effectively, but that’s difficult because the incentives that guide bureaucrats are perverse.
The late, great economist Milton Friedman observed that to gain insight into the motivation of an individual or organization, look for the self-interest. So, where does the self-interest of regulators lie? Not necessarily in serving the public interest, alas, but in expanded responsibilities, bigger budgets, and grander bureaucratic empires for themselves.
The FDA has sometimes exceeded its legislative mandate. Regulators have concocted additional criteria for marketing approval of a new drug above and beyond the statutory requirements, which could inflict significant damage on both patients and pharmaceutical companies. For example, they have arbitrarily demanded that a new drug be superior to existing therapies, although the Food, Drug, and Cosmetic Act requires only a demonstration of safety and efficacy. And Phase 4 (post-marketing) studies are now routine, whereas the FDA used to reserve them for rare situations, such as when there were subpopulations of patients for whom data were insufficient at the time of approval.
The impacts of FDA regulators’ self-serving actions range from the creation of disincentives to research and development (which inflates costs) to significant threats to public health, such as the yearslong delay in the approval of a much-needed meningitis B vaccine .
Another egregious example of the impact of excessive risk-aversion is the sorry saga of a drug called pirfenidone, used to treat a pulmonary disorder called idiopathic pulmonary fibrosis, which used to kill tens of thousands of Americans annually. The FDA unnecessarily delayed approval of the drug for years, although it had already been marketed in Europe, Japan, Canada, and China. During the delay, more than 150,000 patients died of IPF in the United States, many of whom could have benefited from the drug.
A particularly dubious policy is the FDA’s self-declared jurisdiction over “genetically engineered” animals, which virtually destroyed an entire sector of biotechnology. The agency took more than 20 years to approve the first one — an obviously benign, fast-growing salmon — and then botched the five-year review of a single field trial of a genetically engineered mosquito to control the mosquitoes that transmit several viral diseases.
If we are to realize the kind of aggressive innovation-promoting deregulation that the FDA needs, the new commissioner will have to cut the fat and disrupt the agency’s built-in bias for overregulation. We’re not optimistic.