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

Aaron S. Kesselheim; Carolyn L. Treasure; Steven Joffe

doi:10.1371/journal.pmed.1002190

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

Aaron S. Kesselheim ,

* E-mail: akesselheim@partners.org

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

http://orcid.org/0000-0002-8867-2666

⨯
Carolyn L. Treasure,

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
⨯
Steven Joffe

Affiliations 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

http://orcid.org/0000-0002-0667-7384

⨯

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

Aaron S. Kesselheim,
Carolyn L. Treasure,
Steven Joffe

Published: January 3, 2017
https://doi.org/10.1371/journal.pmed.1002190

About the Authors

Aaron S. Kesselheim

* E-mail: akesselheim@partners.org

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

http://orcid.org/0000-0002-8867-2666

Carolyn L. Treasure

Steven Joffe

Affiliations 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

http://orcid.org/0000-0002-0667-7384

Competing Interests

ASK is a member of the Editorial Board of PLOS Medicine.

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Drug research and development
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Oncology
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Point mutation
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Drug therapy
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Non-small cell lung cancer
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Deletion mutation
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Cancer treatment
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Cystic fibrosis
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