Skip to main content

Thank you for visiting nature.com. You are using a browser version with limited support for CSS. To obtain the best experience, we recommend you use a more up to date browser (or turn off compatibility mode in Internet Explorer). In the meantime, to ensure continued support, we are displaying the site without styles and JavaScript.

  • Comment
  • Published:

Clinical pharmacology as a cornerstone of orphan drug development

Nature Reviews Drug Discovery volume 10, pages 795–796 (2011)Cite this article

Subjects

Abstract

A recent US Food and Drug Administration (FDA) advisory committee meeting highlighted the potential of clinical pharmacology to overcome challenges in orphan drug development.

This is a preview of subscription content, access via your institution

Relevant articles

Open Access articles citing this article.

Access options

Buy this article

  • Purchase on Springer Link
  • Instant access to full article PDF
Buy now

Prices may be subject to local taxes which are calculated during checkout

References

  1. Braun, M. M. et al. Emergence of orphan drugs in the United States: a quantitative assessment of the first 25 years. Nature Rev. Drug Discov. 9, 519–522 (2010).

    Article  CAS  Google Scholar 

  2. Tambuyzer, E. Rare diseases, orphan drugs and their regulation: questions and misconceptions. Nature Rev. Drug Discov. 9, 921–929 (2010).

    Article  CAS  Google Scholar 

  3. US FDA. Code of Federal Regulations Title 21: Approval of an Application and an Abbreviated Application. Sec. 314.105 (US Government Printing Office, Washington DC, 2011).

  4. Jadhav, P. et al. Leveraging prior quantitative knowledge in guiding pediatric drug development: a case study. Pharm. Stat. 8, 216–224 (2009).

    Article  Google Scholar 

  5. Bhattaram, V. A. et al. Impact of pharmacometric reviews on new drug approval and labeling decisions — a survey of 31 new drug applications submitted between 2005 and 2006. Clin. Pharmacol. Ther. 81, 213–221 (2007).

    Article  CAS  Google Scholar 

  6. Zhao, P. et al. Application of physiologically based pharmacokinetic (PBPK) modeling and simulation in regulatory review. Clin. Pharmacol. Ther. 89, 259–267 (2011).

    Article  CAS  Google Scholar 

Download references

Author information

Authors and Affiliations

  1. Edward D. Bashaw, Shiew-Mei Huang, Christine E. Garnett, Gilbert Burckart, Lei Zhang, Angela Y. Men, Christine D. Le, Rosane Charlab, and Jogaro V. Gobburu are at the Office of Translational Sciences, Office of Clinical Pharmacology, Center for Drug Evaluation and Research, US Food and Drug Administration, Silver Spring, Maryland 20993, USA.,

    Edward D. Bashaw, Shiew-Mei Huang, Christine E. Garnett, Gilbert Burckart, Lei Zhang, Angela Y. Men, Christine D. Le, Rosane Charlab & Jogaro V. Gobburu

  2. Timothy R. Coté is at the National Organization for Rare Disorders, Washington DC 20008, USA.,

    Timothy R. Coté

  3. Timothy R. Coté is also at the Keck Graduate Institute, Claremont, California 91711, USA.,

    Timothy R. Coté

  4. Anne R. Pariser is at the Office of New Drugs, Center for Drug Evaluation and Research, US Food and Drug Administration, Silver Spring, Maryland 20993, USA.,

    Anne R. Pariser

  5. Lawrence J. Lesko is at the University of Florida, College of Pharmacy, Gainesville, Florida 32610, USA.,

    Lawrence J. Lesko

Authors
  1. Edward D. Bashaw
    View author publications

    You can also search for this author in PubMed Google Scholar

  2. Shiew-Mei Huang
    View author publications

    You can also search for this author in PubMed Google Scholar

  3. Timothy R. Coté
    View author publications

    You can also search for this author in PubMed Google Scholar

  4. Anne R. Pariser
    View author publications

    You can also search for this author in PubMed Google Scholar

  5. Christine E. Garnett
    View author publications

    You can also search for this author in PubMed Google Scholar

  6. Gilbert Burckart
    View author publications

    You can also search for this author in PubMed Google Scholar

  7. Lei Zhang
    View author publications

    You can also search for this author in PubMed Google Scholar

  8. Angela Y. Men
    View author publications

    You can also search for this author in PubMed Google Scholar

  9. Christine D. Le
    View author publications

    You can also search for this author in PubMed Google Scholar

  10. Rosane Charlab
    View author publications

    You can also search for this author in PubMed Google Scholar

  11. Jogaro V. Gobburu
    View author publications

    You can also search for this author in PubMed Google Scholar

  12. Lawrence J. Lesko
    View author publications

    You can also search for this author in PubMed Google Scholar

Corresponding author

Correspondence to Edward D. Bashaw.

Rights and permissions

Reprints and permissions

About this article

Cite this article

Bashaw, E., Huang, SM., Coté, T. et al. Clinical pharmacology as a cornerstone of orphan drug development. Nat Rev Drug Discov 10, 795–796 (2011). https://doi.org/10.1038/nrd3595

Download citation

  • Published:

  • Issue Date:

  • DOI: https://doi.org/10.1038/nrd3595

This article is cited by

Search

Advanced search

Quick links

Nature Briefing: Translational Research

Sign up for the Nature Briefing: Translational Research newsletter — top stories in biotechnology, drug discovery and pharma.

Get what matters in translational research, free to your inbox weekly. Sign up for Nature Briefing: Translational Research