Regulatory red tape threatens survival of rare disease patients

Medical oncologist, geriatrician, and physician scientist GJ van Londen and Chief of Genetic and Genomic Medicine at the University of Pittsburgh School of Medicine Gerald Vockley discuss the article "FDA delays could end vital treatment for rare disease patients." GJ and Gerald explore the complex regulatory impasse where the U.S. Food and Drug Administration denied standard approval for elamipretide despite a positive advisory committee vote, creating a financial crisis that threatens to cut off supply for everyone. GJ shares his personal journey from treating cancer to living with primary mitochondrial myopathy, while the conversation emphasizes the critical need for the agency to use the flexibility granted by the Orphan Drug Act to save a treatment that has already proven its worth. Join us to understand the life-or-death stakes hidden behind administrative decisions.

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