title: Patients as key partners in rare disease drug development.
abstract: Rare disease drug development could benefit substantially from increased patient engagement and input to enhance understanding of the key aspects of disease impact, ways to measure these impacts and patients' perspectives on the benefit-risk profile of potential therapies.
authors.affiliation: Senior Director, Advocacy and Science Policy.
authors.forename: Max G
authors.initials: MG
authors.lastname: Bronstein
authors.affiliation: Everylife Foundation for Rare Diseases, Novato, California 94949, USA.|Ultragenyx, 60 Leveroni Court, Novato, California 94949, USA.
authors.forename: Emil D
authors.initials: ED
authors.lastname: Kakkis
mesh_terms: D002986:Clinical Trials as Topic / Q000379:methods; D055808:Drug Discovery / Q000379:methods*; D006801:Humans; D010358:Patient Participation / Q000379:methods*; D035583:Rare Diseases / Q000175:diagnosis / Q000188:drug therapy*