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Why Patients Are Counting on an FDA Overhaul

President Trump’s “Believe in America” speech to Congress this week has been widely praised by both the Right and Left. Chris Wallace of Fox News even said that “it was one of the best speeches in that setting I’ve ever heard any president give.”

Within that speech, Trump addressed a very unfamiliar topic: orphan drug approvals at the FDA. I don’t recall ever hearing Presidents Obama, Clinton, or either of the Bushes ever mention the FDA, drug pricing, or orphan diseases in an address to Congress.

In honor of Rare Disease Day, Trump invited Megan Crowley to attend his congressional address. Crowley was diagnosed with Pompe Disease, a rare and serious illness, when she was 15 months old. She was not expected to live past age five. Today, she is a college student.

Even more shockingly, while conservatives stood to applause the young woman and Trump addressed drug pricing and rare diseases, the Democrats sternly sat on their hands with unhappy looks on their faces — some of them even shaking their heads in disagreement.

One can only imagine the argument against the development of medications for orphan diseases. I have a feeling Trump could cure cancer and fund the cure-all on his own dime — and liberal naysayers like Sens. Al Franken and Nancy Pelosi would still find something to complain about.

Trump's point is well-wrought. The FDA has badly mismanaged expediting orphan disease drug approvals. I cannot recall any president ever having the courage to mention the FDA to these often overlooked families whose loved ones have rare and orphan diseases. They have fallen through the cracks — while the FDA has had hundreds of employees reviewing "me, too" blockbuster-type drugs.

Related: Your Prescription Isn't Ready Yet [1]

Trump has proposed approving drugs based solely on safety parameters for certain fatal diseases. This might be a great idea, but the FDA would have to do a better job of collecting safety data. As it exists, the FDA only collects between 1 percent and 10 percent of spontaneously reported adverse events. Additionally, it is very important consumers are not gouged on the costs for these experimental drugs as they were Sarepta's exondys51 for Duchenne's muscular dystrophy. In that case, patients were charged $300,000 per year based on poor clinical outcomes.

If people with orphan diseases are volunteering to be guinea pigs to test these drugs, the drugs should be free or nearly free.

In the next several weeks, I hope Trump will announce his pick for FDA commissioner. I believe that conservative candidate Dr. Scott Gottlieb, with his previous drug development and FDA background, is the best choice to "clean house" and lead the restructuring of the FDA.

Dr. David Gortler [2] is a professor of pharmacology and a former Food and Drug Administration senior medical officer; he is now a pharmacology expert [3] and FDA expert with FormerFDA.com. He was the former FDA health care policy adviser for the 2016 Ted Cruz presidential campaign.