title: Introduction to a supplement on innovative approaches to studying health outcomes in rare diseases.
authors.affiliation: From the Program On Regulation, Therapeutics, And Law, Division of Pharmacoepidemiology and Pharmacoeconomics, Department of Medicine, Brigham and Women's Hospital and Harvard Medical School, Boston, MA, USA, akesselheim@partners.org.
authors.forename: Aaron S
authors.initials: AS
authors.lastname: Kesselheim
authors.forename: Joshua J
authors.initials: JJ
authors.lastname: Gagne
mesh_terms: D006801:Humans; D017063:Outcome Assessment, Health Care / Q000379:methods*; D035583:Rare Diseases