title: Participation of patients in the development of advanced therapy medicinal products.
abstract: An increasing number of advanced therapy medicinal products (ATMPs) are under development and in clinical trials. Patients are central to this progress. In research, patients have funded, catalysed, coordinated and led projects. In regulation, patient groups have contributed to the creation of the political momentum for regulation of ATMPs, contributed to the debate and now participate in the regulatory process. Once licensed, patients will have a role in the pharmacovigilance, health technology assessment and reimbursement arrangements for these products. Patient groups contribute valuably as equal stakeholders at every step of the development of an ATMP.
authors.affiliation: EURORDIS (European Organisation for Rare Diseases), Paris, Frankreich.
authors.forename: F
authors.initials: F
authors.lastname: Bignami
authors.forename: A J
authors.initials: AJ
authors.lastname: Kent
authors.forename: M
authors.initials: M
authors.lastname: Lipucci di Paola
authors.forename: N
authors.initials: N
authors.lastname: Meade
mesh_terms: D002986:Clinical Trials as Topic; D003255:Consumer Organizations; D005060:Europe; D015316:Genetic Therapy; D006801:Humans; D010358:Patient Participation; D011057:Politics; D033581:Stem Cell Transplantation; D035703:Therapies, Investigational; D016378:Tissue Transplantation