Abstract
Changes in regulatory policy and legislative incentives to promote the development of drugs for rare diseases — orphan drugs — have led to increases in the number of orphan drug designations, but the rate of such products reaching the market remains frustratingly flat. This article highlights areas in which novel approaches could facilitate regulatory approval and access to treatments for rare diseases.
This is a preview of subscription content, access via your institution
Relevant articles
Open Access articles citing this article.
-
Strategies for eliciting and synthesizing evidence for guidelines in rare diseases
BMC Medical Research Methodology Open Access 28 March 2019
-
Exploring patient and family involvement in the lifecycle of an orphan drug: a scoping review
Orphanet Journal of Rare Diseases Open Access 22 December 2017
-
Review of 11 national policies for rare diseases in the context of key patient needs
Orphanet Journal of Rare Diseases Open Access 31 March 2017
Access options
Subscribe to this journal
Receive 12 print issues and online access
$209.00 per year
only $17.42 per issue
Buy this article
- Purchase on Springer Link
- Instant access to full article PDF
Prices may be subject to local taxes which are calculated during checkout
References
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).
The Committee for Orphan Medicinal Products and the European Medicines Agency Scientific Secretariat. European regulation on orphan medicinal products: 10 years of experience and future perspectives. Nature Rev. Drug Discov. 10, 341–349 (2011).
Acknowledgements
The author would like to acknowledge the GlaxoSmithKline Rare Diseases team who assisted with the preparation of the manuscript and provided helpful comments. Medical writing support was provided by S. Turrell and R. Sherman of Fishawack Communications.
Author information
Authors and Affiliations
Ethics declarations
Competing interests
Marc Dunoyer is an employee of GlaxoSmithKline.
Rights and permissions
About this article
Cite this article
Dunoyer, M. Accelerating access to treatments for rare diseases. Nat Rev Drug Discov 10, 475–476 (2011). https://doi.org/10.1038/nrd3493
Published:
Issue Date:
DOI: https://doi.org/10.1038/nrd3493
This article is cited by
-
Strategies for eliciting and synthesizing evidence for guidelines in rare diseases
BMC Medical Research Methodology (2019)
-
Engagement of Canadian Patients with Rare Diseases and Their Families in the Lifecycle of Therapy: A Qualitative Study
The Patient - Patient-Centered Outcomes Research (2018)
-
Review of 11 national policies for rare diseases in the context of key patient needs
Orphanet Journal of Rare Diseases (2017)
-
Exploring patient and family involvement in the lifecycle of an orphan drug: a scoping review
Orphanet Journal of Rare Diseases (2017)
-
The availability and affordability of orphan drugs for rare diseases in China
Orphanet Journal of Rare Diseases (2016)