Kesselheim, AaronTreasure, CarolynJoffe, Steven2017-02-182017Kesselheim, Aaron S., Carolyn L. Treasure, and Steven Joffe. 2017. “Biomarker-Defined Subsets of Common Diseases: Policy and Economic Implications of Orphan Drug Act Coverage.” PLoS Medicine 14 (1): e1002190. doi:10.1371/journal.pmed.1002190. http://dx.doi.org/10.1371/journal.pmed.1002190.1549-1277http://nrs.harvard.edu/urn-3:HUL.InstRepos:30371184Aaron Kesselheim and colleagues examine orphan-designated drugs approved between 2009 and 2015 in the United States.en-USMedicine and Health SciencesPharmacologyDrug Research and DevelopmentOncologyBiology and Life SciencesGeneticsMutationPoint MutationDeletion MutationCancers and NeoplasmsLung and Intrathoracic TumorsNon-Small Cell Lung CancerPharmaceuticsDrug TherapyDrug ScreeningClinical GeneticsGenetic DiseasesAutosomal Recessive DiseasesCystic FibrosisDevelopmental BiologyFibrosisPulmonologyBiomarker-Defined Subsets of Common Diseases: Policy and Economic Implications of Orphan Drug Act CoverageJournal Article2017-02-1810.1371/journal.pmed.1002190