Thursday, March 02, 2017

Trump used a rare disease survivor to take a shot at the FDA

Julia Belluz · Tuesday, February 28, 2017, 10:22 pm

The president is serious about dismantling the FDA to usher in more medical “miracles.” That’s wrong.

A college student with a rare disease was one of President Donald Trump’s special guests at his address to Congress Tuesday night, which happens to be Rare Disease Day.

Megan Crowley was diagnosed with Pompe, a rare and deadly genetic disorder, when she was 15 months old. Doctors gave her six months to live.

Instead of accepting Megan’s fate, her father, John, founded a company to search for a cure. The company, Novazyme Pharmaceuticals, eventually created an enzyme replacement therapy for Pompe, which Crowley’s son was also later born with. The Crowley children are still treated with it today, according to Stat News. (The family was profiled in the book The Cure, which inspired the 2010 Harrison Ford movie Extraordinary Measures.)

Tonight, Trump used the Crowleys’ story to take a dig at the Food and Drug Administration, which he’s been lambasting for its sluggishness for the past several months.

“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,” he said. “If we slash the restraints, not just at the FDA but across our government, then we will be blessed with far more miracles just like Megan.”

Unfortunately, that’s not how it works. According to drug industry experts, and those who study the FDA, the agency isn’t what gets in the way of drug innovation.

It’s not the FDA that gets in the way of innovation — it’s the limits of our knowledge

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