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Accelerating Hope: The Pathway for Cures (Redux)

Accelerating Hope: The Pathway for Cures (Redux)

When people were dying from AIDS in the 1980s and 90s, Congress found a way to expedite the drug approval process and saved countless lives. Established in 1992, the Accelerated Approval Program allows the FDA to speed approval of drugs for patients with serious and life-threatening conditions. In this episode we talk with the father of children with a rare disease, a company CEO working to treat rare diseases and two policy experts on Accelerated Approval.

I AM BIO · John Crowley Executive Chairman Amicus Therapeutics, Emil D. Kakkis CEO and President Ultragenyx, Cartier Esham Chief Scientific Officer BIO, Duane Schulthess CEO Vital Transformation

July 4, 202323m 0s

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Show Notes

When people were dying from AIDS in the 1980s and 90s, Congress found a way to expedite the drug approval process and saved countless lives. Established in 1992, the Accelerated Approval Program allows the FDA to speed approval of drugs for patients with serious and life-threatening conditions. In this episode we talk with the father of children with a rare disease, a company CEO working to treat rare diseases and two policy experts on Accelerated Approval.

Topics

aidscartier eshamrare cancermsclinical benefitkeri russellsickle cell diseasepatientsalzheimer’sbiobrendan frasercmsultragenyxjohn crowleyemil kakkisharrison forddrug approvalrare diseasesaccelerated approvaldrug policyvital transformationexpedited drug approvalcenters for medicare and medicaid servicestreatmentssurrogate endpointduane schulthessamicus therapeuticspompe diseasenew drugsultra-rare diseasemuscular dystrophyextraordinary measureshivbiomarkershep cfda