title: Biomarker-Defined Subsets of Common Diseases: Policy and Economic Implications of Orphan Drug Act Coverage.
abstract: Aaron Kesselheim and colleagues examine orphan-designated drugs approved between 2009 and 2015 in the United States.
authors.affiliation: Program On Regulation, Therapeutics, And Law (PORTAL), Division of Pharmacoepidemiology and Pharmacoeconomics, Department of Medicine, Brigham and Women's Hospital and Harvard Medical School, Boston, Massachusetts, United States of America.
authors.forename: Aaron S
authors.identifier: http://orcid.org/0000-0002-8867-2666
authors.initials: AS
authors.lastname: Kesselheim
authors.affiliation: Program On Regulation, Therapeutics, And Law (PORTAL), Division of Pharmacoepidemiology and Pharmacoeconomics, Department of Medicine, Brigham and Women's Hospital and Harvard Medical School, Boston, Massachusetts, United States of America.
authors.forename: Carolyn L
authors.initials: CL
authors.lastname: Treasure
authors.affiliation: Department of Medical Ethics & Health Policy, University of Pennsylvania Perelman School of Medicine, Philadelphia, Pennsylvania, United States of America.|Leonard Davis Institute of Health Economics, University of Pennsylvania, Philadelphia, Pennsylvania, United States of America.
authors.forename: Steven
authors.identifier: http://orcid.org/0000-0002-0667-7384
authors.initials: S
authors.lastname: Joffe
mesh_terms: D015415:Biomarkers / Q000032:analysis; D017277:Drug Approval / Q000191:economics / Q000331:legislation & jurisprudence; D006291:Health Policy; D006801:Humans; D009965:Orphan Drug Production / Q000191:economics / Q000331:legislation & jurisprudence; D035583:Rare Diseases / Q000188:drug therapy; D014481:United States; D014486:United States Food and Drug Administration
chemical_list: D015415:Biomarkers