The FDA has released draft guidance to simplify the approval process for personalized gene therapies, a move that rare disease advocates have called for. This draft guidance, published on Tuesday, aims to address the lengthy and expensive approval process, which has led to delays in treatment and limited access for patients. Paul Scribner, vice president of community programs and education at the PKD Foundation, expressed optimism about the draft guidance, saying it "puts a flag in the ground and says, 'This is important.'"
Key points
The FDA's move is expected to be a win for rare disease advocates, who have long called f…: The FDA's move is expected to be a win for rare disease advocates, who have long called for more streamlined and accessible treatments for patients with rare genetic disorders.
The draft guidance aims to address the lengthy and expensive approval process, which has…: The draft guidance aims to address the lengthy and expensive approval process, which has led to delays in treatment and limited access for patients.
Paul Scribner, vice president of community programs and education at the PKD Foundation,…: Paul Scribner, vice president of community programs and education at the PKD Foundation, expressed optimism about the draft guidance, saying it "puts a flag in the ground and says, 'This is important.'"
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WASHINGTON – The Food and Drug Administration published draft guidance on Tuesday that would streamline approvals of some individualized gene therapies, a win for rare disease advocates who have criticized the agency’s approach to these treatments.
“It’s sort of putting a flag in the ground and saying, ‘This is important,’” Paul Scribner, vice president of community programs and education at the PKD Foundation, said of the draft guidance at a rare disease event on Tuesday.
Breakthroughs in gene therapies, or treatments that treat or cure diseases by altering faulty genes inside a person’s cells, have energized researchers and companies who believe many patients with rare mutations could benefit from these personalized medicines.
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