Rare Disease R&D

Our founder Julie Walters speaks to Contract Pharma about finding the hard-to-reach and leveraging big data and machine learning in drug development

Rare disease research and development has grown significantly these past several years, fueled by innovation, and leveraging the FDA's Orphan Drug Designation, which offers sponsors certain incentives. In 2019, 21 of the FDA’s 48 novel drug approvals (44%) were Orphan Drugs. The Orphan Drug Act provides special status to a drug or biologic to treat a rare disease or condition affecting fewer than 200,000 Americans. Orphan designation qualifies the sponsor for various development incentives, including tax credits for qualified clinical testing, waived prescription drug user fees, and eligibility for seven years of marketing exclusivity.

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