5 things to know about the $300k rare disease drug praised by Trump
Here are five things to know.
1. During his speech, the president acknowledged Rare Disease Day. He shared the story of 20-year-old Megan Crowley who has a rare, lifethreating condition called Pompe disease.
2. Megan's father — John Crowley, current CEO of Amicus Therapeutics — founded a biotech company in 1998 to develop a treatment for Pompe disease.
"Megan's dad, John, fought with everything he had to save the life of his precious child. He founded a company to look for a cure and helped develop the drug that saved Megan's life," President 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."
3. The Food and Drug Administration approved the first treatment for Pompe in 2006, with a second similar drug approved in 2010. Sanofi, who now owns both the drugs, said the average cost of treatment is $298,000, reports Reuters.
4. The National Organization for Rare Disorders commended President Trump's focus on rare disease, but said it does not share his views that the FDA needs a regulatory overhaul, according to the report.
"Our patients deserve the same quality therapies as everyone else, and to weaken the standards will only threaten our population with unsafe, ineffective therapies," NORD said Wednesday in a statement.
5. Aaron Kesselheim, MD, a researcher at Harvard Medical School in Boston, shared similar skepticism.
"I think Trump used this example somewhat cynically as a way of trying to advance the deregulatory agenda," he told Reuters. "The FDA's standards actually promote innovation because patients don't want just any old drug - they want drugs that work."
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