New opportunities for accessing promising non-oncological orphan drugs - Carla E.M. Hollak; Carelle C. Reparon-Schuijt; Kathelijn Verdeyen; Lisa J. Deesker; Jaap W. Groothoff; Josephine M.I. Vos; Atse H. Huisman; Silvia van der Flier; Anke A.M.G. Pisters-van Roy (The Lancet Regional Health. Europe)