For anyone strongly concerned that the Priority Review Voucher scheme won’t be renewed, it’s worthwhile considering the following:The Give Kids a Chance Act of 2024, which authorized the FDA to continue awarding PRVs through to September 2029, was passed unanimously in September by a Republican-controlled Congress. The Act had been introduced by a Republican Congressman and was cosponsored by both Democrats and Republicans. This Act now needs to pass the Senate, which has now changed from Democrat to Republican control.
Separately, the Creating Hope Reauthorization Act of 2024 has been introduced into the Senate by both a Democrat and a Republican. This Act proposes that the FDA be authorized to continue awarding PRVs through to September 2030.
Further, for anyone who is fearful that a Donald Trump administration might lead to a hardened stance by the FDA towards rare disease, here is what Trump said in an address to Congress in early 2017, shortly after being sworn in for his first term as President
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 5.
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.
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