Tuesday night, in his address to Congress, President Trump invited as his guest a college sophomore with a rare disease to illustrate why the Food and Drug Administration needs to be ripped to pieces. After 20-year-old Megan Crowley was diagnosed with the neuromuscular disorder Pompe disease as a young child, her father helped found a biotech company to develop a drug to save her.
“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 in his address. “If we slash the restraints, not just at the FDA but across our government, then we will be blessed with far more miracles like Megan.”
Crowley’s story is well known: it was the subject a 2010 Hollywood drama starring Harrison Ford and Keri Russell called Extraordinary Measures. The thing is, the FDA’s regulatory process is not what stood in the way of developing a drug to save Megan Crowley—a lack of basic scientific knowledge was. Nor would dismantling the agency have brought that drug to market more expeditiously.
There is actually a lot of interest within the pharmaceutical industry in developing so-called ‘orphan drugs’ to treat diseases affecting fewer than 200,000 people, like Pompe disease. That’s because in 1983, Reagan signed into law the Orphan Drug Act, creating significant financial incentives for the development of orphan drugs. While orphan drugs still have to go through the same FDA evaluation process used for other new drugs, the FDA is typically more flexible in evaluating treatments for rare disease. In fact, the drug Crowley’s father helped to develop for Pompe disease, Myozyme, was approved in under a year based on trials of just 39 people.
That’s not unusual. Citing a report from the National Organization for Rare Disorders, the FDA said that about two-thirds of orphan drugs are approved with one good clinical trial, whereas two or three clinical trials are typically required for mass market drugs. Approvals for new orphan drugs have more than doubled over the past decade, with a record 37 of them approved in 2015.
Still, Trump has repeatedly called out the FDA for its slow approval process, beyond just rare diseases. His general thinking in this arena is that there are just too many regulations, and those regulations stand in the way of bringing new drugs to market quickly. In January, he told a group of pharmaceutical company executives that he planned to slash more than 75 percent of drug regulations. “Instead of it being 9,000 pages, it’ll be 100 pages,” he said.
But in reality, the FDA isn’t all that slow. It aims to approve most drugs in under 10 months, and priority drugs in less than six, making it among the fastest drug approval processes in the world. Review times for new drugs have steadily been decreasing in recent decades, dropping from 30 months in the 1980s to 8.5 months today.
Trump also criticized high drug prices, a talking point that has generated much praise in light of recent price gouging fiascos like the EpiPen. On Tuesday, he told lawmakers that he would “work to bring down the artificially high price of drugs and bring them down immediately.”
It’s true that programs like the Orphan Drug Act, which incentivizes drug development in part by offering developers a longer exclusive license on their inventions, have resulted in drugs that cost more than people think they should. Trump didn’t elaborate on how he plans to reform those high drug prices. But here, too, it’s not roadblocking regulations that are the problem. Last year, the FDA approved 800 new generic drugs for market, more than it ever had in a single year. Those drugs will likely lower costs for patients, providing less expensive alternatives to name-brand therapies and introducing the competition Trump keeps talking about into the market.
Trump has been mum on the details of exactly how all of this radical deregulation might happen, nor has he nominated a commissioner to head the FDA. One of his potential nominees, however, is Silicon Valley investor Jim O’Neill, an outspoken critic of FDA regulations. “Let’s prove efficacy after they’ve been legalized,” O’Neill has said of new drugs.
Despite what O’Neill may think, there ample evidence that the drug approval process, at least, is working just fine. Some in the pharmaceutical industry, in fact, have pushed back against deregulation. They worry that a less strict drug approval process would result in drugs insurers and patients are unwilling to pay for without sufficient proof that they’ll actually work.
In the mean time, one thing sure to bring more drugs to market: more funding for scientific research. It’s hard to develop a new drug for a disease when you don’t have enough information on the disease. The drug that Jim Crowley developed to help save his daughter moved through the FDA swiftly—at the time, in fact, it was heralded as a great success of the Orphan Drug Act. Where Crowley and his company ran into trouble was in raising funds to study and treat a disease few people had ever heard of.