Next Generation Orphan Drug Incentives

4:30 PM - 5:30 PM, Tuesday, June 9, 2020 ・ Room 10
Powered by private sector incentives, the Orphan Drug Act became law in 1983 and has been a phenomenal public health success resulting in hundreds of therapies for unmet needs. Today, the promise of gene and cell therapies, advances in genetic screening and related diagnostics, and precision medicine has generated widespread enthusiasm throughout the rare disease community. At the same time, new patient-focused drug development is giving patient groups a pathway to new treatments. Yet, despite advances, 95% of rare diseases still do not have a treatment. Are the existing Orphan Drug Act incentives aligned with current science and patient-focused drug development? With Congress considering ways to update the 21st Century Cures Act, and new studies imagining future scenarios, this session will explore new approaches to incentives and patient collaboration to foster a new era in orphan drug development for the 30 million Americans suffering with rare disease.