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.

  • Commentary
  • Published:

Accessing the accelerated approval pathway for rare disease therapeutics

Nature Biotechnology volume 34, pages 380–383 (2016)Cite this article

Subjects

Improvements must be made to the qualification process for biomarkers as primary endpoints in pivotal clinical studies of treatments for the rarest of diseases.

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

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

Figure 1

References

  1. FDA. Accelerated approvals. http://www.fda.gov/Drugs/DevelopmentApprovalProcess/HowDrugsareDevelopedandApproved/DrugandBiologicApprovalReports/NDAandBLAApprovalReports/ucm373430.htm (accessed 31 December 2014 and 31 December 2015).

  2. Kakkis, E.D. et al. Orphanet J. Rare Dis. 10, 16 (2015).

    Article  Google Scholar 

  3. Miyamoto, B.E. & Kakkis, E.D. Orphanet J. Rare Dis. 6, 49 (2011).

    Article  Google Scholar 

  4. US Food and Drug Administration. Center for Drug Evaluation and Research. Guidance for Industry. Expedited Programs for Serious Conditions-Drugs and Biologics. (FDA, Washington, DC, 2014). http://www.fda.gov/RegulatoryInformation/Guidances/default.htm (accessed 7 July and 23 August 2014).

  5. Sasinowski, F.J. Drug Info. J. 46, 238 (2012).

    Article  Google Scholar 

  6. FDA. New drugs at FDA: CDER's new molecular entities and new therapeutic biological products. http://www.fda.gov/Drugs/DevelopmentApprovalProcess/DrugInnovation/default.htm (accessed July 7, 2014 and January 15, 2015).

  7. Waisbren, S.E. et al. Mol. Genet. Metab. 92, 63–70 (2007).

    Article  CAS  Google Scholar 

  8. Food and Drug Administration Safety and Innovation Act (FDASIA), Pub. L. 112–144, 126 Stat. 993, codified as amended at 21 U.S.C. §301 (2012).

  9. FDA. Rare Diseases: Common Issues in Drug Development Guidance for Industry. http://www.fda.gov/downloads/Drugs/GuidanceComplianceRegulatoryInformation/Guidances/UCM458485.pdf (accessed January 18 2015).

Download references

Author information

Authors and Affiliations

  1. EveryLife Foundation for Rare Diseases, Novato, California, USA

    Emil D Kakkis, Sara Kowalcyk & Max G Bronstein

Authors
  1. Emil D Kakkis
    View author publications

    You can also search for this author in PubMed Google Scholar

  2. Sara Kowalcyk
    View author publications

    You can also search for this author in PubMed Google Scholar

  3. Max G Bronstein
    View author publications

    You can also search for this author in PubMed Google Scholar

Corresponding author

Correspondence to Emil D Kakkis.

Ethics declarations

Competing interests

E.K. is a current employee of and stockholder in Ultragenyx, a stockholder in BioMarin and president of the EveryLife Foundation for Rare Diseases.

Supplementary information

Supplementary Information

Supplementary Tables 1 and 2 (DOCX 48 kb)

Rights and permissions

Reprints and permissions

About this article

Check for updates. Verify currency and authenticity via CrossMark

Cite this article

Kakkis, E., Kowalcyk, S. & Bronstein, M. Accessing the accelerated approval pathway for rare disease therapeutics. Nat Biotechnol 34, 380–383 (2016). https://doi.org/10.1038/nbt.3530

Download citation

  • Published:

  • Issue Date:

  • DOI: https://doi.org/10.1038/nbt.3530

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