Will the Trump presidency mean the end of FDA drug regulation?

By Michael HiltzikBusiness Columnist

Nov. 16, 2016 3:14 PM PT

Donald Trump will be coming into office waving the banner of deregulation. While most of the speculation about his plans has focused on the financial industry and the possibility of eviscerating Dodd-Frank reforms, keep your eyes on the Food and Drug Administration. In a Trump administration the agency, figuratively speaking, will have a big bull’s-eye on its back.

For decades, the FDA has been a target of conservatives and libertarians, who assert that it routinely blocks potentially life-saving drugs by demanding too much pre-market testing. Trump didn’t talk much about this during the campaign, but he picked up the cudgel after election day, declaring as part of his healthcare policy the intention to “reform the Food and Drug Administration, to put greater focus on the need of patients for new and innovative medical products.”

As governor of Indiana his vice president-elect, Mike Pence, signed a “right-to-try” law, which absolves drug makers of legal responsibility for hawking untested nostrums at terminally ill patients.

Supporters of the idea that drugs should be tested for efficacy before being sold are in for a long four years.

— Douglas Sipp, Riken Center for Developmental Biology

Oncologist and quack-buster David Gorski calls such state laws a “cruel sham” — first because states have no authority to allow drugs unapproved by the FDA, and because a federal version would “eliminate … the FDA’s ability to protect terminally ill patients from what could be dangerous or inappropriate drugs.” They sound good, but they are not innocuous.

The goal of libertarians — and now presumably Trump — is to lower or even eliminate barriers to marketing whatever drug companies wish, unless they’re proven to be dangerous. This would open the door to a torrent of expensive treatments that aren’t shown to be better than less expensive drugs already on the market, or even to have any effect at all.

“Supporters of the idea that drugs should be tested for efficacy before being sold are in for a long four years,” comments Douglas Sipp of the Riken Center for Developmental Biology in Kobe, Japan. Who will pay for the reduction of efficacy standards? You will, through higher health insurance premiums and higher taxes to pay for drugs prescribed for patients on Medicare and Medicaid … drugs not shown to work.

None of this is to say that the FDA works perfectly in all cases. The agency currently is trying to get its arms around a mandate from Congress that it take patients’ concerns better into account when pondering whether to approve experimental treatments for rare diseases with no known alternative cures.

“Most patients in that situation would like to gamble,” USC health economist Darius Lakdawalla told me recently. “They’d like to swing for the fences.” For them, the FDA’s insistence on extensive clinical trials to establish safety and efficacy is callous and inexplicable.

“It’s OK to have different standards” for untreatable conditions and for diseases with abundant treatments, Lakdawalla says. “The question becomes, how different ought those standards to be? What’s the value of a drug with uncertain prospects to patients without alternatives?”

The difficulty is knowing where to draw the line — how far to allow standards to slip to allow an unproven treatment into the marketplace. That very question deeply fractured the FDA staff over the last few months, as we reported in October.

The issue was the agency’s approval of Exondys 51, a drug to treat Duchenne muscular dystrophy, despite what staff scientists considered to be an almost total absence of clinical evidence that the drug works. To them, this wasn’t even a close call. Based on clinical tests of the drug, the FDA record described its effect on production of the protein needed by patients to counteract the disease variously as “minuscule,” “a mere scintilla” and “not perceptibly greater than none.” Moreover, FDA reviewers observed, the tests themselves were unreliable.

Nevertheless, approval was pushed through almost single-handedly by Janet Woodcock, a senior official at the agency, who cited her own “medical/scientific judgment” in defending her decision. This drew a retort from FDA Acting Chief Scientist Luciano Borio that “approving products based on hope, on subjective clinical judgment, or on theoretical constructs that are not anchored in data leads to irreparable damage to patients.”

Borio argued that relaxing standards to approve Exondys 51 could “make matters worse for patients with no existing meaningful therapies” by discouraging further work on other treatments and allowing a rush of untested and potentially dangerous drugs for other diseases into the market. FDA staff also decried pressure tactics from patient advocates, including abusive correspondence. That’s a sign of the emotion that often accompanies appeals for accelerated approval of drugs for desperate patients.

FDA officials say its policy on accelerated approval for drugs aimed at “unmet medical needs” is evolving. But it’s complicated by the agency’s authority to give a drug thumbs-up or thumbs-down treatment, nothing in between. The agency should concern itself primarily with “keeping unsafe drugs off the market,” argues USC’s Lakdawalla. “If a drug’s not unsafe but there are questions about its effectiveness, they ought to err on the side of approval.”

Yet sometimes evidence of harm comes slowly, by which point it may be too late for many patients. Merck’s anti-inflammatory drug Vioxx was marketed with FDA approval for five years before evidence that it contributed to heart attacks and stroke prompted its withdrawal in 2004. And the agency’s cautious approach to the morning sickness drug thalidomide in the 1960s may have prevented birth defects in thousands of children of women who could have taken it during pregnancy. Families in Europe, where it was a popular drug, weren’t so lucky.

And back in 1994, Sens. Orrin Hatch (R-Utah) and Tom Harkin (D-Iowa), served their corporate patrons by sponsoring a bill to remove dietary supplements largely from the FDA’s jurisdiction. The harvest has been the almost totally unrestricted marketing of adulterated and hazardous nostrums by an industry now bloated to over $32 billion a year in sales.

Conservatives in Congress have consistently tried to strip the FDA of even more regulatory authority despite these object lessons. They’re abetted by libertarian propaganda that blames the agency for killing people by taking a careful approach to ostensibly life-saving drugs. But this position requires accepting drugmakers’ claims for their own products as gospel, hard evidence be damned.

Who will watch out for the public’s safety once the FDA is scared away? Trump’s assertion that patients “need … new and innovative medical products” is true but incomplete. He left out the part about “new and innovative medical products” that work. Will that standard still be part of the FDA’s charter in the new administration?

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