1:45 PM (PT)
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Clinical trials in small populations, particularly those with progressive or degenerative rare diseases, face scientific and ethical hurdles that challenge traditional trial frameworks. High phenotypi ...Breakout Session
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Orphan and Rare Disease
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3:00 PM (PT)
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Clinical trials have too often failed to represent the communities they aim to serve - limiting the relevance, reach, and real‑world impact of medical innovation. The Robert A. Winn Excellence in Clin ...Breakout Session
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Workforce, Health Equity, and Leadership
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4:15 PM (PT)
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Congress created the Rare Pediatric Disease Priority Review Voucher (PRV) program to incentivize the development of drugs for serious or life-threatening rare diseases in children. Vouchers are earned ...Breakout Session
- Orphan and Rare Disease
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We are living in a golden age of biopharmaceutical innovation. Yet, too often, patients face barriers between them and the appropriate treatment they need across various cost-shifting programs. Payers ...Breakout Session
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Patient Advocacy
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1:45 PM (PT)
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In recent years, a confluence of global economic, market, and policy shifts have led to the abandonment of more than 1,000 promising treatments in development for rare diseases - many of which deliver ...Breakout Session
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Business Development and Investment
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