President Donald Trump outlined his political agenda for 2017 in his address to a joint session of Congress Tuesday night, aiming a broadside directed straight at the “burdensome” drug approval process at the FDA. And a prominent CEO in the rare disease field stepped into Trump’s high powered spotlight to urge a change in the way the agency handles orphan drugs while making a pitch for a speedy reversal of a painful setback handed to him by regulators last year.
In a series of pointed highlights during tonight’s speech, most of Trump’s comments on healthcare focused on the now standard attack on Obamacare and a demand to repeal and replace his predecessor’s plans on health insurance. But he reserved a few moments of heated criticism reserved specifically for the FDA and government reform.
“We must eliminate the burdensome approval process for life-saving drugs so that more lives can be saved,” was included in Trump’s advance list of talking points.
“(O)ur slow and burdensome approval process at the FDA keeps too many advances, like the one that saved Megan’s life, from reaching those in need,” Trump told his audience, turning to Amicus CEO John Crowley’s daughter Megan in the audience.
Megan Crowley suffers from Pompe disease, a rare ailment that Amicus has set out to treat with two drugs now in Phase I. Crowley founded Novazyme, acquired by Genzyme, which went on to develop an enzyme replacement therapy Megan Crowley still uses.
“If we slash the restraints, not just at the FDA but in government,” Trump told Congress, “we will be blessed with far more miracles like Megan.”
Earlier in the speech Trump also lashed out once again at the “artificially high price of drugs,” which he has vowed to rein in.
That all fits closely with the president’s insistence — outlined in a recent session with Big Pharma executives — that the drug approval process needs to be deregulated, insisting that slashing FDA rules was needed in order to cut drug prices. But the industry has recoiled at the idea that the FDA’s rules governing development need to be gutted, leaving many wondering just how extreme a makeover the president has in mind.
Amicus $FOLD represents a biotech company which would benefit significantly from a sharp change in direction at the FDA. Last year the company’s shares were sent into a tailspin after it was forced to mount a new safety study for migalastat, a new drug for Fabry disease, after regulators at the FDA demanded more data in a move that will push back any approval by years. Months before, the European Commission gave it a green light across the Atlantic.
“(I)t is troubling that in recent years, rare disease research is becoming what the Orphan Drug Act sought to change: an enterprise too expensive to justify investment,” the Amicus CEO wrote in a piece for the Observer. “It takes now orphan drugs as much time to complete Phase III studies (the stage of drug development where efficacy is established) as it does for non-orphans. At the same time, the average clinical trial cost per patient is 14 times higher for orphan drugs compared to those that are not. The FDA’s regulation of the orphan development process is becoming less flexible, less efficient and less patient-centered.”
“Similarly,” he added, “my company, Amicus Therapeutics, saw the development of Galafold, for the treatment of Fabry disease, slowed by the same process in the U.S., adding millions of dollars in cost and delaying treatment for years, despite the drug’s approval as a precision medicine in Europe last year. The FDA has declared that the only pathway to availability for Fabry patients in the United States is predicated on yet another study. And while we do that, patients wait. All get sicker. And some will die.”
Investors picked up on the possibilities, bidding Amicus’ shares up 7% Wednesday morning.
Now the biotech industry wants to know if a new FDA commissioner appointed by Trump would change the balance of safety and efficacy data needed for an approval, changes which may benefit individual companies but erode the gold standard long demanded by the agency.
Crowley has already made it clear that he believes he’s ready to start marketing the drug in the US “today.” His fate this year will influence the entire industry, for better or worse, as Trump and the nation’s lawmakers go about transforming the president’s rhetoric into political reality.
Trump mischaracterizes Pompe drug approval process. Approved in 9 months based on 39 patients. Not "slow and burdensome."
— David A. Kessler MD (@DavidAKesslerMD) March 1, 2017
Remarks As Delivered:
An incredible young woman is with us this evening who should serve as an inspiration to us all.
Today is Rare Disease day, and joining us in the gallery is a Rare Disease Survivor, Megan Crowley. Megan was diagnosed with Pompe Disease, a rare and serious illness, when she was 15 months old. She was not expected to live past five.
On receiving this news, 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. Today she is 20 years old — and a sophomore at Notre Dame.
Megan’s story is about the unbounded power of a father’s love for a daughter.
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.
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.
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