Is the Trump Administration the Cure the FDA Needs?
It was exhilarating in December when the Senate joined the House to demonstrate that Congress can accomplish something. With overwhelming bipartisan support, both passed the 21st Century Cures Act. Donald Trump's repeated chastisement of the FDA during the campaign for over-regulation and not doing enough to promote cures was also encouraging.
Finally, a Congress and president who see pharma as a resource and not a villain. Though in recent weeks, Trump has demonized drug pricing, which resulted in a decline in industry stock values.
Of course, critics such as Bernie Sanders (I-VT) and Elizabeth Warren (D-MA) voted against the act, which they called a boondoggle that will benefit the pharma and biotech industries while endangering patients. They think the act's provisions, which includes allowing increased use of biomarkers, are dangerous shortcuts. For them, when it comes to drug approval, the more glacial the better.
What Sanders and Warren don't get is the purpose of the act is not to hamstring the FDA, but to rein it back to its original purpose. Instead of providing basic efficacy and safety guidance, the agency has become a bureaucratic quagmire trying to predict long-term treatment outcomes while factoring drug pricing into the approval process. Moreover, as Dr. Joseph Gulfo noted in The Wall Street Journal, the FDA has increasingly begun to ignore the advice of its experts.
I've witnessed this first-hand. In 2010, the FDA was considering the NDA of Esbriet, a breakthrough treatment for idiopathic pulmonary fibrosis (IPF), a disease with mortality rates higher than most cancers. Under another name, Esbriet had been approved in Japan. Two large, double-blind U.S. and European studies had shown the drug significantly slowed disease progression. After reviewing the data, the FDA's Pulmonary-Allergy Advisory Committee voted 9-3 in favor of approval. Was this good enough for the FDA?
Nope. A few days later, the FDA second-guessed its own committee and insisted on more data. The manufacturer was forced to undertake yet another enormously expensive double-blind study.
Shortly after the FDA's rejection, Esbriet was approved by Europe's NICE board on the basis of the same data. Result? Americans with IPF who wanted a proven treatment were forced to travel to Japan or Europe. Esbriet was eventually approved in 2014, but the human cost of the four-year delay was inexcusable.
Medical innovation isn't easy. For many diseases, the science just isn't there. As recent studies have shown, we're not sure what even causes Alzheimer's. What's more, developing a drug can take more than 10 years and cost more than $1 billion. The economic burden has forced major companies such as Pfizer, Merck, and Lilly to downsize and rely on startup biotechs for R&D.
At the same time, funding for the National Institutes of Health has been cut in federal budget after budget.
The 21st Century Cures Act is a great start because it sends nearly $5 billion to the NIH to support programs such as the Cancer Moonshot, the Precision Medicine initiative, and brain research. I hope it works.
But perhaps we can do more. Michael Milken writing in The Wall Street Journal threw out this challenge: “Why don't we, in the private economy, take advantage of the fresh wind in Washington and offer to partner with the new leadership to advance our nation's health? It could be huge — hopefully!”
Sander Flaum is a principal at Flaum Navigators.