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jeff stier

Yes, The FDA Is In Trouble, But The New York Times’ Prescription Would Make It Worse

The FDA’s failures are a result of too much regulatory dithering and bureaucracy. Yet the Times’ prescriptions for change would just increase the dose.

A recent New York Times editorial about the Food and Drug Administration (FDA) reflects a systematic weakness at the once-venerable Gray Lady: The members of the editorial board often rely on sloganeering and popular wisdom instead of substantive evidence.

The editorial was headlined, “The FDA Is in Trouble. Here’s How to Fix It.” The agency is in trouble. But it’s due to the very kinds of “fixes” the Times recommends.

The FDA is highly bureaucratic and risk averse, leading to a slow and expensive drug approval process—at last count, more than $2.5 billion to bring a new drug to market. Yet the armchair quarterbacks at the Times want to slow it down even more and increase the cost and risks of innovating.

Supposedly, too many drugs are being approved “with too little data on how safe or effective they are,” according to the editorial. More specifically, regulators have supposedly made “compromises” by accepting “surrogate evidence” of efficacy.

In fact, there are good reasons that the clinical testing of new drugs can be accomplished with fewer and smaller trials. We are entering the era of precision, or personalized, medicine, the mantra of which is “the right dose of the right drug for the right patient at the right time.”

It reflects that treatments are gradually shifting from a relatively imprecise one-size-fits-all approach to a more personalized one, so patients can be matched to the best therapy based on their genetic makeup, the specific characteristics of their illness, and other predictive factors. This enables doctors to avoid prescribing a medication that is unlikely to be effective or that might cause serious side effects in certain patients.

How Smaller Trials Can Be More Accurate

The editorial ignores that those factors make possible drug testing in smaller, better-targeted populations. That is not a completely new concept. Under appropriate circumstances, the FDA has long used fewer and smaller clinical trials as the basis for approval.

What makes that possible is that medical research is increasingly discovering biological indicators, or “biomarkers”—such as variants of DNA sequences, the levels of certain enzymes, or the presence or absence of drug receptors—that can dictate how patients should be treated and to predict the likelihood that the intervention will be effective or elicit dangerous side effects.

Using biomarkers enables drug companies to better select patient populations for clinical trials to demonstrate efficacy. The reason is related to the statistical power of clinical studies: In any kind of experiment, a fundamental principle is that the greater the number of subjects or iterations, the greater the confidence in the results. Conversely, small studies generally have large uncertainties about results—and that is where biomarkers can make a difference.

By better defining the experimental groups, such as limiting the trial only to patients with a certain mutation in their genome or tumor, they can help drugmakers design clinical studies that will show “a high relative treatment difference” between the drug and whatever it is being compared to (often a placebo, but sometimes another treatment).

For example, a 2018 study of patients with certain rare pancreatic or gastrointestinal cancers found that analyzing the “protein-signaling networks” in the tumors could identify regulators of tumor survival. The researchers were then able to test the effect of various drugs on these regulators. That enabled them to predict in many patients which drugs would be effective in the tumors—the kind of precision oncology that makes possible smaller clinical trials.

Whiffing on Needed Critiques of the FDA

The Times editorial faulted the FDA for “its roles in the opioid epidemic (regulators allowed too many opioids on the market without properly flagging them as addictive or deadly),” but, in fact, the regulators did ensure that the drugs were safe and effective when used according to the labels, which do, in fact, warn about addiction potential. Analogously, can the Bureau of Alcohol, Tobacco, Firearms and Explosives be blamed for many Americans suffering from alcoholism?

Criticizing the FDA for its handling of e-cigarettes is easy. But the Times editorial even got that wrong, echoing the calls of prohibitionists to ban the sale of these products to adult smokers, rather than aggressively enforcing the existing ban on sales to minors.

The Times could have landed a powerful science-based critique of the agency for perpetuating the activist-created myth that nicotine e-cigarettes had anything to do with the past year’s lung disease outbreak, which was caused by adulterated THC oils, not nicotine vapes. By incorrectly blaming e-cigarettes for the illnesses, the FDA’s misinformation prevented countless adult smokers from switching to a truly less harmful alternative. The Times failed to hold the agency accountable for not telling the truth when it mattered most.

The Times editorial accuses the agency of having become “too susceptible to outside pressure,” which most FDA-watchers find to be groundless. If FDA has favored any special interests, they are “progressive” ones, including the organic food industry, which has systematically violated regulations concerning “absence claims” on labels (such as “GMO free”), and by acceding to the demands of “public health advocates” who reject harm reduction policies toward e-cigarettes. The remedy for such failings is better, smarter management.

The FDA Needs a Diet, Not More Money

The Times editorial claims the FDA “has too few resources and too little power to fulfill its key responsibilities.” The facts argue otherwise. According to the Congressional Research Service:

Between FY2015 and FY2019, FDA’s enacted total program level increased from $4.507 billion to $5.725 billion. Over this time period, congressionally appropriated funding increased by 21%, and user fee revenue increased by 35%. The Administration’s FY2020 budget request was for a total program level of $5.981 billion, an increase of $256 million (+4%) over the FY2019 enacted amount ($5.725 billion).

More important than the raw numbers is how FDA’s resources are being used. The agency has become extremely top-heavy, with ever more boxes appearing at the top of the organizational chart, even though the vast majority of day-to-day oversight and regulatory actions are taken at the level of FDA’s various “centers”—the Center for Drug Evaluation and Research, Center for Food Safety and Nutrition, and so on. The FDA needs to be put on a diet, not to have additional “resources.”

The Times editorial endorsed a recent proposal to convert FDA from a component of the Department of Health and Human Services to an independent agency. That would be a prescription for disaster. Political meddling with the agency’s decisions has been extremely rare in recent years, and the genuine calamities in which the FDA has been involved have been self-inflicted wounds that might have been avoided with more, not less, accountability and oversight.

Three distinguished former federal officials presented in the journal Health Affairs compelling procedural arguments against making FDA an independent agency. As an independent agency, they said, FDA would not be bound by the policies of the Department of Justice, potentially leading to inconsistent positions being taken by different parts of the government on issues that could include foreign policy.

Furthermore, they point out, applying a consistent approach to rulemaking as required by working within Health and Human Services and Office of Management and Budget strictures is a useful check on highly expensive or wrong-headed regulation. “It’s also a way that Congress and the president can ensure consistency across government in the application of expertise in regulatory policy,” they wrote.

The FDA’s failures are a result of too much regulatory dithering and bureaucracy. Yet the Times’ prescriptions for change would just increase the dose.

Originally published here.


The Consumer Choice Center is the consumer advocacy group supporting lifestyle freedom, innovation, privacy, science, and consumer choice. The main policy areas we focus on are digital, mobility, lifestyle & consumer goods, and health & science.

The CCC represents consumers in over 100 countries across the globe. We closely monitor regulatory trends in Ottawa, Washington, Brussels, Geneva and other hotspots of regulation and inform and activate consumers to fight for #ConsumerChoice. Learn more at 
consumerchoicecenter.org

The Deep State Will Challenge the New FDA Head

FDA

If we are to realize the kind of aggressive, innovation-promoting deregulation called for by President Trump, Stephen Hahn (FDA) will need to disrupt the agency’s built-in bias for overregulation.

Now that the Trump Administration’s new FDA commissioner, Dr. Stephen Hahn, has been confirmed, he’ll find he has one of the most difficult and important jobs in government. The FDA’s purview is wide, regulating pharmaceutical and other medical, food, and vaping products that account for more than 25 cents of every consumer dollar, over a trillion dollars annually.

Government regulation offers some reassurance to the public, to be sure, but when it is wrong-headed or merely fails to be cost-effective, it actually costs lives—directly by withholding life-saving and life-enhancing products, and also indirectly by diverting societal resources to gratuitous regulatory compliance.

Dr. Hahn is inheriting an organization that is huge, critical, and dysfunctional. The stakes are high. For example, 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 will never be developed at all.

Putting FDA on the right track will require toughness and discipline at an agency where more than 99.9 percent of the employees are civil servants who cannot be fired even for incompetence or insubordination. (Did we hear someone mutter, “deep state?”)

Government regulators have vast power and wide discretion; unfortunately, the incentives that guide them 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.

Former FDA Commissioner Frank E. Young once quipped that “dogs bark, cows moo, and regulators regulate.” Consistent with that propensity, FDA has sometimes exceeded its congressional mandate. Regulators have concocted additional criteria for marketing approval of a new drug—above and beyond the statutory requirements for demonstrating safety and efficacy—that 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 (postmarketing) studies are now routine, whereas the FDA used to reserve them for rare situations, as when there were subpopulations of patients for whom data were insufficient at the time of approval.

The effects of FDA regulators’ self-serving actions range from the creation of disincentives to research and development (which inflates their costs) to significant threats to public health, such as the years-long delay in 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 (IPF), 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.

Many years of fat budgets have enabled the FDA to waste resources. In 2017, for example, the agency sought public comments about its use of focus groups, claiming they “provide an important role in gathering information because they allow for a more in-depth understanding of patients’ and consumers’ attitudes, beliefs, motivations, and feelings.” FDA officials seem to have forgotten that their mission is to make science-based decisions—primarily about product safety, efficacy, and quality—as expeditiously as possible, whatever the public’s beliefs, motivations, and feelings may be.

A particularly dubious policy is the FDA self-declared jurisdiction over all “genetically engineered” animals. Subsequently, the agency then took more than 20 years to approve the first one—an obviously benign, fast-growing salmon—and then made a colossal mess of the five-year review of a single field trial of a mosquito to control the mosquitoes that transmit the Zika, yellow fever, dengue fever, and chikungunya viruses. Eventually, the FDA relinquished jurisdiction over that mosquito and other animals with pesticidal properties to the EPA, where they belong.

We need structural, policy, management, and cultural changes that create incentives for FDA to regulate in a way that is evidence-based and imposes the minimum burden possible. A number of possible approaches and remedies to accomplish that have been described, ranging from radical to more conservative.

Significant legislative changes, or even meaningful congressional oversight, would go a long way toward reining in an agency so culturally invested in more regulation. But political realities make this unlikely anytime soon.

If we are to realize the kind of aggressive, innovation-promoting deregulation called for by President Trump, Hahn will need to disrupt the agency’s built-in bias for overregulation.

Originally published here


The Consumer Choice Center is the consumer advocacy group supporting lifestyle freedom, innovation, privacy, science, and consumer choice. The main policy areas we focus on are digital, mobility, lifestyle & consumer goods, and health & science.

The CCC represents consumers in over 100 countries across the globe. We closely monitor regulatory trends in Ottawa, Washington, Brussels, Geneva and other hotspots of regulation and inform and activate consumers to fight for #ConsumerChoice. Learn more at 
consumerchoicecenter.org

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