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

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

Aaron S Kesselheim  1 , Carolyn L Treasure  1 , Steven Joffe  2   3
Affiliations

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

Aaron S Kesselheim et al. PLoS Med. .

Abstract

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

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Conflict of interest statement

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

Figures

Fig 1
Fig 1. Orphan drug designations per year.
Fig 2
Fig 2. Monthly cost of three subgroups of oncologic drugs.
See S1 Appendix for raw data used in these figures.
See this image and copyright information in PMC

References

    1. Orphan Drug Act of 1983. 21 U.S.C. 360bb(a)(2) (2008). https://www.gpo.gov/fdsys/pkg/STATUTE-96/pdf/STATUTE-96-Pg2049.pdf Accessed 2 Sept 2016.
    1. Reardon S. Regulators adopt more orphan drugs. Nature. 2014;508:16–17. 10.1038/508016a - DOI - PubMed
    1. Field MJ, Boat TF. Committee on Accelerating Rare Diseases Research and Orphan Product Development Board on Health Sciences Policy. Washington, DC: National Academies Press; 2010. - PubMed
    1. Herder M. When everyone is an orphan: against adopting a U.S.-styled orphan drug policy in Canada. Account Res. 2013;20(4):227–69. 10.1080/08989621.2013.793120 - DOI - PubMed
    1. Daniel MG, Pawlik TM, Fader AN, Esnaola NF, Makary MA. The Orphan Drug Act: Restoring the Mission to Rare Diseases. Am J Clin Oncol 2015;00(00):1 http://content.wkhealth.com/linkback/openurl?sid=WKPTLP:landingpage&an=0... - PubMed

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