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

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

PLoS Med. 2017 Jan 3;14(1):e1002190. doi: 10.1371/journal.pmed.1002190. eCollection 2017 Jan.

Authors

Aaron S Kesselheim¹, Carolyn L Treasure¹, Steven Joffe^{2

3}

Affiliations

¹ 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.
² Department of Medical Ethics & Health Policy, University of Pennsylvania Perelman School of Medicine, Philadelphia, Pennsylvania, United States of America.
³ Leonard Davis Institute of Health Economics, University of Pennsylvania, Philadelphia, Pennsylvania, United States of America.

Abstract

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

MeSH terms

Biomarkers / analysis
Drug Approval* / economics
Drug Approval* / legislation & jurisprudence
Health Policy*
Humans
Orphan Drug Production* / economics
Orphan Drug Production* / legislation & jurisprudence
Rare Diseases / drug therapy
United States
United States Food and Drug Administration

Substances

Biomarkers

Grant support

ASK's work is supported by the Laura and John Arnold Foundation, with additional support from the Engelberg Foundation. ASK is also a Greenwall Faculty Scholar and is supported by the Harvard Program in Therapeutic Science. The funders had no role in study design, data collection and analysis, decision to publish, or preparation of the manuscript.