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EFPIA Challenges and facilitators in the development of orphan and paediatric medicines

The European Federation of Pharmaceutical Industries and Associations (EFPIA) is the trade association representing the biopharmaceutical industry in Europe. Is has direct membership of 37 national associations, 38 leading pharmaceutical companies and a growing number of small and medium-sized enterprises (SMEs). EFPIA’s mission is to create a collaborative environment for its members to innovate, discover, develop, and deliver new therapies and vaccines for people across Europe.

This report, developed collaboratively by EFPIA and the Office of Health Economics (OHE), presents a conceptual model for understanding how to best ensure sustained innovation in areas of need. It also provides illustrative case studies on five different therapies to highlight specific challenges and facilitators around developing medicines for rare and paediatric conditions.

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Challenges and facilitators in the development of orphan and paediatric medicines

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