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Biomarker-Defined Subsets of Common Diseases: Policy and Economic Implications of Orphan Drug Act Coverage

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2017

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Public Library of Science
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Kesselheim, 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.

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Abstract

Aaron Kesselheim and colleagues examine orphan-designated drugs approved between 2009 and 2015 in the United States.

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Medicine and Health Sciences, Pharmacology, Drug Research and Development, Oncology, Biology and Life Sciences, Genetics, Mutation, Point Mutation, Deletion Mutation, Cancers and Neoplasms, Lung and Intrathoracic Tumors, Non-Small Cell Lung Cancer, Pharmaceutics, Drug Therapy, Drug Screening, Clinical Genetics, Genetic Diseases, Autosomal Recessive Diseases, Cystic Fibrosis, Developmental Biology, Fibrosis, Pulmonology

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