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Advances in identifying the genetic causes of diseases, and particularly cancers, coupled with regulatory incentives to encourage the development of therapeutic approaches to once poorly managed orphan diseases have fueled a surge in exciting new medicines. However, developing such drugs requires new approaches designed to tackle some of the challenges associated with rare diseases.

To address some of these issues and explore potential solutions, Simbec-Orion in collaboration with Informa Pharma Intelligence convened a round table of experts attending the BIO International Convention. A consistent recommendation from the panel, which had representatives from organizations associated with different stakeholders in orphan drug development, was better and sustained engagement with patients.

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