High anxiety at FDA over leadership, faster drug and medical device oversight

To hear some powerful proponents tell it, Uncle Sam needs to really hurry up the government’s approval of drugs and medical devices. He’s got to make oversight over them easier, lighter, and less complex.

But consider just some of the health news in recent days:

• A “weak reporting system” allowed doctors and hospitals to escape for years reporting cases in which a tissue-shredding surgical tool used to operate on the uterus spread cancer cells throughout women’s bodies, The New York Times says. This worsens the disease and dims patients’ survival chances. It was not until 2013, however, that the government, prodded by reports in the Wall Street Journal and other media, collected “adverse incident” data on the tool, called a morcellator, and women’s cancers. By 2016, regulators had amassed reports of 258 women with cancers tied to morcellation procedures—and some of these cases already have proved fatal.
• Investigators re-examining a critical component of dozens of infections and deaths tied to diagnostic medical scopes found that the devices often are damaged in ways that prevents them from being cleaned thoroughly, even with new measures instituted after a national scandal involving them, the Los Angeles Times reports.
• New research shows that 72 cancer-fighting therapies, which regulators raced to market between 2002 and 2012, didn’t benefit patients as much as hoped, giving them just 2.1 months more life than existing medications. That’s according to a Kaiser Health News story carried by CNN. It also reports that a researcher who studies oncology medications says two-thirds of cancer drugs approved in the last two years have shown no evidence they extend patients’ lives. Meantime, a group of academic researchers has called for a halt to medicine’s proliferating $100,000-a-year and more cancer medications, arguing that there are better ways to develop effective drugs without the astronomic costs that make them unaffordable except to a very few.
• Outrage is growing over regulators’ expedited, swift approval of deflazcort as an “orphan” drug (a medication targeted to treat a disease afflicting only a small number of patients) for Duchene muscular dystrophy. This inherited condition afflicts 1 in 3,600 boys, and because it worsens rapidly, few survive into their 20s. Questions have arisen about this drug and its effectiveness with Duchene’s, according to a trade industry publication’s report. Marathon, the drug’s maker, plans to charge $89,000 annually for it—and that is what has caused experts to sputter. Deflazcort, a simple steroid, has been on the market for decades, and is sold overseas for as little as $1 per dose. But a company acquired rights to the drug, pushed a small study that showed it appeared to boost Duchene’s patients strengths, and, suddenly the renamed medication Emflaza has been designated an exclusive therapy for a rare pediatric disorder, a valuable economic designation.

What’s the inescapable diagnosis about the federal Food and Drug Administration, specifically, and U.S. oversight, generally, on prescription drugs and medical devices? It’s flawed, and it desperately needs its own care and considerable attention. This is no secret. The recently passed, $6.3 billion 21^st Century Cures Act funds and pushes for this reform, perhaps giving too much leeway, I have written, to Big Pharma and big medical device makers.

Off-kilter approach to dire needed change

I’ve also written about the Trump Administration’s off-kilter approach, thus far, to the FDA. With the advice of Peter Thiel, a Silicon Valley entrepreneur, the president has talked with venture capitalists with little deep experience in medical science or health care about heading the FDA. One of the reported finalists for this mission-critical post is a self-avowed libertarian who has said that he knows of no patients who are forced to take a drug or to use a medical device, so, they should do so at their own risk, with little government oversight such as rigorous tests for safety and effectiveness.

The administration, with the help of the majority GOP on the Hill, is slowly filling its top posts. And the anxiety about the FDA’s future and the administration choice to lead it has become palpable among medical and public health experts, as well as leaders in Big Pharma and device makers, too.

The Los Angeles Times, in a Sunday page one story, used the $94,000-a-year cancer drug Tarceva as an example of what can go awry when drugs get raced through a regulatory process. The newspaper says that a 2010 decision by the FDA expanded use of the drug, “despite warnings from a panel of experts who said there was little evidence it actually worked.” As the Los Angeles Times reported:

The FDA’s decision resulted in more harm than the hundreds of millions of dollars wasted on a drug that was ineffective for 90 percent or more of patients.  Over the years, thousands of patients were prescribed a drug that had no chance of helping them but could frequently cause terrible rashes covering their faces and bodies, according to prescription data and adverse event reports. Some patients with the most aggressively growing cancers died before trying other treatments that may have lengthened their lives.

In my practice, I certainly see the terrible harms that dangerous drugs and defective devices can cause. I’m glad that the media keep pointing out problems with bad drugs and medical devices. I’m pleased to see lawmakers like Sen. Chuck Grassley (R.-Iowa) investigate Big Pharma and orphan drugs. I’ve written about these price-gouging practices.

I’ve also written how the president hasn’t inspired great public confidence that he knows how to improve regulatory oversight over prescription drugs, especially with their soaring prices, and medical devices. With Tweets and broad statements, he suggests he gets the significance of problems in this sector. But then he backs away, or undercuts his pronouncements with countervailing deed or statement.

Make no mistake about it: Soaring drug costs are one of the big drivers of the nation’s constantly rising health care expenses, and Americans are both terrified by this and sick of it. Crushed by the cost of drugs and medical services, many are postponing needed care because they can’t afford it, especially if they have health insurance but are saddled with high-deductibles that they must pay out of their own pocket first. More than a quarter of Americans know someone in their family who is struggling with medical debt, which I consider unconscionable. Medical debt is the No. 1 cause of bankruptcy.

We all admire Silicon Valley’s transformational innovation, and how technology has improved our lives. But when it comes to our health, and critical components of modern caregiving like drugs and medical devices, none of us wants to be a guinea pig for wealthy entrepreneurs wishing to test their economic theories and political ideologies.