How Advanced Therapies are Changing the Landscape of Rare Disease
February 28, 2019 - Toronto ON CA

Phone: 1 416.977.6555

Join experts from Medpace’s medical, regulatory and operational team with hands-on experience in rare disease and ATMP development as they explore: Scientific Advancements: The new and exciting landscape for advanced therapies in rare disease for a variety of indications, with a deeper dive into hematology and ophthalmology Regulatory Landscape: Key characteristics and considerations for obtaining orphan drug development (ODD) designation in the US as well as an overview of the comparative differences among current global orphan product laws and regulations Operational Considerations: Lessons-learned and best practices for operationalizing rare disease clinical trials including identifying, enrolling and retaining patients, as well as special considerations for ATMPs

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