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

This report summarises the contents of an EFPIA workshop which gathered Senior staff from member company R&D, Clinical, Strategy, and Commercial functions to tackle the issue of unmet medical needs in rare and paediatric diseases. It first describes an explanatory framework that illustrates how biopharmaceutical companies make investment decisions, taking into account scientific, commercial, and policy factors. Applying this framework, the report then examines the hurdles that have impeded innovation in two ‘white spots’ where limited treatments are available: extremely rare diseases and paediatric-onset diseases. Finally, this report outlines ways forward towards lowering unmet needs in those white spots.