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.


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


  • Biomarkers

Grants and funding

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.