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. This episode is presented by Scholar Advising, a fee-only financial advising firm specializing in providing advice for DIY investors. If you want clear, actionable strategies and confidence that your financial decisions are built on objective advice without AUM fees or commissions, Scholar is designed for you. Physicians often navigate complex compensation structures, including W-2 income, 1099 work, production bonuses, and practice ownership. Scholar's highly credentialed advisors guide high-earners through decisions like optimizing investments for long-term tax efficiency and expert strategies for financial independence. Every recommendation is tailored to the financial realities physicians face. VISIT SPONSOR → https://scholaradvising.com/kevinmd SUBSCRIBE TO THE PODCAST → https://www.kevinmd.com/podcast RECOMMENDED BY KEVINMD → https://www.kevinmd.com/recommended