Time for Change? The Why, What and How of Promoting Innovation to Tackle Rare Diseases - Is It Time to Update the EU's Orphan Regulation? And if so, What Should be Changed?

Horgan, Denis; Moss, Barbara; Boccia, Stefania; Genuardi, Maurizio; Gajewski, Maciej; Capurso, Gabriele; Fenaux, Pierre; Gulbis, Beatrice; Pellegrini, Mariangela; Del Mar Mañú Pereira, Maria; Gutiérrez Valle, Victoria; Gutiérrez Ibarluzea, Iñaki; Kent, Alastair; Cattaneo, Ivana; Jagielska, Beata; Belina, Ivica; Tumiene, Birute; Ward, Adrian; Papaluca, Marisa

doi:10.1159/000509272

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Time for Change? The Why, What and How of Promoting Innovation to Tackle Rare Diseases - Is It Time to Update the EU's Orphan Regulation? And if so, What Should be Changed?

Shared by NobleBlocks on Sep 30, 2026 • 08:58 PM UTC

Authors:

Denis Horgan

Barbara Moss

Stefania Boccia

Abstract

Since developments are global in the healthcare arena, more should be done to align EU and other big markets' regulatory practices for rare disease patients. Notwithstanding efforts and cooperation between the US and EU aimed to harmonize their strategic plans in the field of orphan drugs, regulator...

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