President Trump on Tuesday spent a portion of his speech to a joint session of Congress airing his grievances about prescription drugs, both in how they’re approved and how much they cost.
Trump reiterated his interest in bringing down drug prices, telling Congress he wants to “work to bring down the artificially high price of drugs … immediately.”
Trump singled out Megan Crowley – a 20-year-old living with Pompe disease, a rare inherited disorder that leads to a buildup of a complex sugar in organs and tissues – to talk about the regulatory process at the FDA.
Shortly after she was diagnosed, Megan Crowley’s father, John, started working to find a treatment, and Megan entered a clinical trial by 2003. John is the CEO of rare-disease company Amicus Therapeutics. The company’s share price jumped 12% Wednesday after the presidential shout-out.
“But our slow and burdensome approval process at the Food and Drug Administration keeps too many advances, like the one that saved Megan’s life, from reaching those in need,” Trump said. “If we slash the restraints, not just at the FDA, but across our government, then we will be blessed with far more miracles just like Megan.”
Not everyone agreed that the rare-disease drug was approved slowly. Dr. David Kessler, who served as FDA commissioner from 1990 to 1997, had a different take:
Trump mischaracterizes Pompe drug approval process. Approved in 9 months based on 39 patients. Not “slow and burdensome.”
— David A. Kessler MD (@DavidAKesslerMD) March 1, 2017
Regardless of how fast the particular drug was approved, Trump’s anecdote is in line with his plans to deregulate the drug industry. “We’re going to be cutting regulations at a level that nobody’s ever seen before,” Trump said in a meeting with pharma executives on January 31. He estimated up to 80% of regulations would be slashed.
The FDA is responsible for regulating food and drugs. It’s also responsible for regulating medical devices, blood donations, veterinary products, cosmetics, and tobacco. Trump’s comments have left the drug industry concerned about what that deregulation could mean for the drug-approval process.
Here are some examples of the FDA at work
- It, of course, approves new drugs. In 2016, 22 new drugs made the cut, compared to 45 the year before. In September 2016, the FDA banned antibacterial soap based on evidence that showed that the soaps weren’t any better, cleaner, or safer than regular soap. The agency cracked down in April 2016 when diet supplements showed up containing a dangerous stimulant. The FDA keeps track of any side effects that come up after a drug’s approved, and can make changes accordingly. In July, the agency changed the label on a commonly used class of antibiotics to reflect more potentially permanent side effects. It makes sure ads for drugs aren’t misleading.
The FDA was officially founded in 1906, when the Food and Drugs Act was signed into law, prohibiting misbranded food, drinks, and drugs from interstate commerce. This cracked down on misleading claims that treatments could “cure” patients.
Following the thalidomide birth-defect crisis that happened in western Europe, the FDA got more leeway to make sure drugs were not just safe but effective. That’s been the standard for the past 55 years, but it’s possible that Trump’s pick will be interested in changing back the regulation to make it just revolve around safety.
While Trump still hasn’t disclosed his FDA commissioner nod, there are three reported candidates:
Jim O’Neill, managing director at Mithril Capital, a libertarian who has said he’s in favor of approving drugs that are safe, before they’re shown to be effective. Dr. Scott Gottlieb, a resident fellow at the American Enterprise Institute and former deputy commissioner of the FDA during George W. Bush’s presidency. Gottlieb has emerged as a favorite among drug executives. Dr. Joseph Gulfo, a former biotech executive who advocates for a color-coded approval system.
What the drug industry thinks about the FDA
In theory, getting more drugs approved at a faster pace would seem like a good idea. Drug development from start to finish can often take longer than a decade, and it can be an expensive process.
But the process also builds up evidence that the drug companies can point to show that their drugs work they way they’re approved to. That can help get the drugs paid for by insurance plans. So far, the response from industry executives and experts has been against cutting regulations that would make the FDA approve drugs solely based on their safety.
“The underpinnings of belief among patients, payers, even investors, is that somebody out there has tested these things and has shown, with some evidence, that they work,” Harvard professor Daniel Carpenter told The New York Times.
There’s a fair amount of concern that by making sure drugs are just safe, and not necessarily effective, the US might start to have more “snake oil” treatments.
“Without rigorous scientific and medical proof to establish that a drug is both Safe and Effective, we risk taking a step back into a time when people sold colored water for cancer treatments and patients became the unwitting tools of unscrupulous marketeers,” Ovid Therapeutics CEO Jeremy Levin told Endpoints in February.