March 1, 2017
By Alex Keown, BioSpace.com Breaking News Staff
WASHIGNTON – During his first address to a joint session of Congress, President Donald Trump once again decried what he called a “slow and burdensome” process of drug approval in the United States.
The address to Congress took place on Rare Disease Day, a day typically used to lobby policy makers and the public about the impact of rare diseases and raise awareness about therapeutic research. During the speech, Trump introduced Megan Crowley, a college student at the University of Notre Dame, who was diagnosed with Pompe disease at 15 months old. Pompe disease is an enzyme deficiency that leads to muscle and respiratory issues in patients. When Crowley was diagnosed with Pompe, her doctors gave her six months to live. But, Trump said her father, John Crowley, didn’t accept that diagnosis and founded a company, Novazyme Pharmaceuticals, to search for a cure. Researchers at Novazyme developed an enzyme replacement therapy that has allowed Megan and her brother, who was also diagnosed with Pompe, to live long past their initial life expectancy. Novazyme was acquired by Genzyme in 2001.
Trump hailed John Crowley’s innovation that led to the saving of his children’s lives. Trump said if Congress supports the slashing of restraints at the U.S. Food and Drug Administration as well as other government agencies, “then we will be blessed with far more miracles like Megan,” Trump said, according to STAT.
This is not the first time Trump has called for deregulating the FDA. Throughout the presidential campaign, Trump said he intended to “cut the red tape” at the FDA. According to Trump’s 100 Day Plan, there are “over 4,000 drugs awaiting approval, and we especially want to speed the approval of life-saving medications.” Trump has also called for easing restrictions for drugs that have been approved by foreign regulatory agencies to be available in the United States, which would bypass the FDA’s regulatory authority over drugs available in the U.S.
Trump has yet to appoint a new head of the FDA. Several of the candidates who have been floated as possible contenders are seen as disruptors, particularly Jim O’Neill, who has a history of calling for reforms at the FDA, including allowing for the approval of drugs after they have been proven safe, but have not yet necessarily proven efficacy. Trump has also floated Dr. Scott Gottlieb, a former deputy commissioners at the FDA. Gottlieb has strong ties to the pharmaceutical industry and currently serves as an adviser to several companies, including GlaxoSmithKline . Unlike O’Neill and others, Gottlieb is not seen as a disruptor by the healthcare industry.
During Tuesday night’s address, Trump also stressed his hope to dismantle the Affordable Care Act. He announced the intention to introduce a plan to replace the ACA and bring down healthcare costs and the “artificially high price of drugs.”
Trump has been harshly critical of the drug industry, saying the companies were “getting away with murder” when it comes to the prices the companies charge for medication. Shortly after he took office, Trump met with industry leaders and said competition was the key to lowering drug prices and said he would oppose regulations that “makes it harder for smaller, younger companies to take the risk of bringing their product to a vibrantly competitive market.” Trump said he would oppose “price fixing by the biggest dog in the market, Medicare.”
