title: Accelerating development, registration and access to medicines for rare diseases in the European Union through adaptive approaches: features and perspectives.
abstract: There is growing recognition that the current research-and-development (R&D) and innovation-regulation ecosystem could be made more efficient to stimulate and support access to innovative therapies for those patients with rare, life-threatening diseases for which there are no adequate licensed therapies. New and progressive thinking on the principles and processes of drug development and regulation are needed in rare disease settings in order to ensure developments are financially sustainable. This paper presents perspectives on the current and emerging schemes for accelerating development of and access to medicines for rare diseases in the European Union.
authors: Voisin Consulting Life Sciences, Paris, France. uguen@voisinconsulting.com.
authors: David
authors: D
authors: Uguen
authors: Thomas
authors: T
authors: Lönngren
authors: Yann
authors: Y
authors: Le Cam
authors: Sarah
authors: S
authors: Garner
authors: Emmanuelle
authors: E
authors: Voisin
authors: Carlo
authors: C
authors: Incerti
authors: Marc
authors: M
authors: Dunoyer
authors: Moncef
authors: M
authors: Slaoui
mesh_terms: D005062:European Union; D006801:Humans; D009965:Orphan Drug Production; D035583:Rare Diseases