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The backstory behind the rare disease patient Trump highlighted to Congress

  • Author: Carolyn Y. Johnson, The Washington Post
  • Updated: February 28, 2017
  • Published February 28, 2017

Midway through his address to Congress, President Donald Trump paused to highlight the inspiring story of Megan Crowley, 20, whose life is a testament – in part – to American entrepreneurship.

When Megan was diagnosed with an extremely rare disease as a baby, her father, John Crowley, founded a small biotechnology startup to try to cure Megan and her brother – an effort that ultimately grew into an 80-person company and helped create the drug that keeps Megan healthy today.

Megan's affliction, Pompe disease, is exceedingly rare. The course of the disease varies by patient, but it causes children's muscles to weaken and, eventually, their lungs may fail. Today, thanks to the work of her father – and the American biotechnology industry – Megan is a sophomore at University of Notre Dame.

Guest Megan Crowley watches President Donald Trump’s first address before a joint session of Congress. Must credit: Washington Post photo by Jonathan Newton

Trump said that cutting regulations at the Food and Drug Administration, which regulates drugmakers, would be a path to more stories like Megan's.

"If we slash the restraints, not just at the FDA but across our government, then we will be blessed with far more miracles like Megan," Trump said. "In fact, our children will grow up in a nation of miracles."

Crowley's story is gripping enough that it led to a book and inspired a 2010 movie, "Extraordinary Measures," starring Harrison Ford as the father. And in many way it seems to hit all the right notes for Trump's vision of American business and medicine: John Crowley wasn't an industry insider. When he founded his first biotech company, he was motivated by the desire to cure his kids, not pharmaceutical greed. He was a tough, driven American dad who created American jobs.

But pull back the curtain a little further, and it becomes clear that this success story is quintessentially American in a different way than Trump might care to admit. The actual drug that saved Megan's life is manufactured in Belgium and was developed by a biotech company founded by a Dutch immigrant – a company that is today owned by a French firm. The drug was invented through a scientific experiment that couldn't have happened without international collaboration. And a president who has said he wants to bring down drug prices just held up as a shining example of innovation a drug that costs an average of $298,000 a year, per patient.

It's also unclear whether the Crowley's inspiring story and the development of the drug Myozyme (also called Lumizyme) is really an example of how "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," as Trump described.

Other companies have pointed to Myozyme as an example of the agency's flexibility in getting drugs approved quickly, based on small amounts of data.

Briefing materials prepared by the drug company Sarepta Therapeutics to support the approval of their drug last year cited Myozyme as an "approval precedent" that they hoped to follow.

Last April, at a hearing in support of Sarepta's drug, one of the researchers involved in Myozyme's approval testified to the advisory committee that its passage through the regulatory process as a helpful parallel to consider.

Against the recommendation of its advisory committee, which wanted to see more evidence, regulators approved that drug.

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