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The backstory behind the rare disease patient Trump highlighted to Congress
WASHINGTON, DC - FEBRUARY 28: People applaud Megan Crowley, 20, during, President Donald J. TrumpÕs first address before a joint session of Congress on Tuesday, Feb. 28, 2017, at the US Capitol in Washington, DC. (Photo by Melina Mara /The Washington Post)
People applaud Megan Crowley. (Photo by Melina Mara/The Washington Post)

Midway through his address to Congress, President 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 start-up 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.

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, where he was played by Brendan Fraser. 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.

Correction: A previous version of this post incorrectly stated which actor played John Crowley in the movie, Extraordinary Measures. He was played by Brendan Fraser.

Real-time fact-checking and analysis of Trump’s address to Congress
WASHINGTON, DC - FEBRUARY 28: U.S. President Donald Trump addresses a joint session of the U.S. Congress as Vice President Mike Pence (L) and House Speaker Rep. Paul Ryan (R) (R-WI) look on on February 28, 2017 in the House chamber of the U.S. Capitol in Washington, DC. Trump's first address to Congress focused on national security, tax and regulatory reform, the economy, and healthcare. (Photo by Chip Somodevilla/Getty Images)
Trump addresses a joint session of Congress on Tuesday. (Photo by Chip Somodevilla/Getty Images)

President Trump is headed to the Capitol tonight to deliver his first speech as president to a joint session of Congress. The speech is expected to outline budget goals and priorities.

The speech is scheduled to start at 9 p.m.; you can watch coverage starting at 8:45 p.m. here.

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