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.

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