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Addressing unmet needs in extremely rare and paediatric-onset diseases: how the biopharmaceutical innovation model can help identify current issues and find potential solutions

Emilie Neez, Arianna Gentillini, Adam Hutchings
July 2021

Although the Orphan and Paediatric Regulations have been successful at stimulating innovation in these previously underserved areas, significant unmet medical needs remain. EFPIA held in May 2021 a workshop, which gathered Senior staff from member company R&D, Clinical, Strategy, and Commercial functions, to identify barriers to addressing unmet medical needs and potential ways to overcome them. Considering that science, economics, and policy coalesce to inform investment decisions, it is clear that these three dimensions must be addressed in concert for innovation to flourish in extremely rare and paediatric-onset diseases.

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