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President Donald Trump characterized the FDA as having a “slow and burdensome approval process” that keeps lifesaving treatments from patients.
Trump’s comments, made Feb. 28 in his first joint address to Congress, indicate the agency may be facing regulatory changes, especially coming just four days after his executive order on regulatory reform.
One of Trump’s reported picks to lead the Food and Drug Administration, Silicon Valley investor Jim O’Neill, has suggested the agency should focus solely on new drugs’ safety, leaving the market to decide whether they work.
“If we slash the restraints, not just at the FDA but across our government, then we will be blessed with far more miracles,” Trump said.
The speech coincided with Rare Disease Day, and the president invited Megan Crowley, who has the rare neuromuscular disorder Pompe disease and whose story inspired the movie Extraordinary Measures, to the joint session. Crowley’s father John, frustrated with the pace of research in the late 1990s, joined bioetchnology company Novazyme Pharmaceuticals (later acquired by Sanofi Genzyme) and credits an experimental trial with saving his children’s lives.
“Megan’s story is about the unbounded power of a father’s love for a daughter,” Trump said. “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.”
Research!America President Mary Woolley said the president’s “moving” remarks about Crowley highlight the need to ensure that the FDA and other health agencies have the resources they need to make the discovery, development and delivery of new medicines a reality.
“It is also fundamentally important to maintain rigorous safety and efficacy standards to ensure the treatments Americans receive live up to their promise,” Woolley said. “With robust funding for research and smart policies to further drive public and private sector innovation, we can help Megan and millions of others realize their dreams.”
“It’s all about the money,” Carol Pratt, an FDA regulatory attorney with Lee & Hayes PLLC, told Bloomberg BNA in a March 1 email.
Pratt said Trump’s comments on the FDA are fully aligned with the focus of the 21st Century Cures Act ( Pub. L. 114-255), a sweeping bipartisan bill to spur new medical treatments. But the big unknown is how Trump’s $54 billion diversion of funding from federal programs to the military will affect Cures Act implementation, especially because many of the law’s provisions don’t have secure funding.
“Accelerating innovating treatments does not mean the FDA can sit on its hands and do nothing,” Pratt said. “It means the agency has to develop alternative [approval] pathways and that is not budget neutral.”
Michael Carome, director of Public Citizen’s Health Research Group, said Trump’s characterization of the FDA “reflects complete ignorance about the FDA’s current regulatory schemes for ensuring that medications and medical devices are safe and effective.”
“The regulatory pendulum for the FDA’s review of medical products already has swung too far toward promoting speed over the rigor of evidence demonstrating safety and effectiveness,” Carome said in a March 1 statement.
“Trump’s reckless proposal to slash FDA regulations in an effort to further speed FDA’s review of medical products would dangerously undermine FDA’s approval processes and lead to countless preventable injuries, deaths and illnesses across the U.S.”
The agency’s leaders repeatedly have rejected the FDA’s image as a stodgy agency that takes too long to approve new medicines.
Peter Lurie, associate FDA commissioner for public health strategy and analysis, said at a Senate hearing last September that more than half of the new drugs approved over the past several years came out in the U.S. first.
“That is a very old story and frankly an outdated story,” Lurie said at the hearing.
The FDA approves more novel drugs per year than its European and Japanese counterparts, according to a study released a few years ago. The agency’s own 2016 report concluded 19 of the 22 novel drugs approved in 2016, or 86 percent, were approved in the U.S. before any other country.
The agency accounts for a relatively small piece of the estimated 10-year, $2.58 billion price tag associated with bringing a new drug to market. Most of those costs are generally attributed to complex, expensive clinical trials, and because most new drug compounds fail. The FDA estimates that only five out of 5,000 drug compounds that enter preclinical testing ever advance to clinical trials.
To contact the reporter on this story: Jeannie Baumann in Washington at firstname.lastname@example.org
To contact the editor responsible for this story: Randy Kubetin at RKubetin@bna.com
A transpcript of Trump's remarks is at http://src.bna.com/mD7.
Copyright © 2017 The Bureau of National Affairs, Inc. All Rights Reserved.
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