Skip to main page content
U.S. flag

An official website of the United States government

Dot gov

The .gov means it’s official.
Federal government websites often end in .gov or .mil. Before sharing sensitive information, make sure you’re on a federal government site.

Https

The site is secure.
The https:// ensures that you are connecting to the official website and that any information you provide is encrypted and transmitted securely.

Access keys NCBI Homepage MyNCBI Homepage Main Content Main Navigation
. 2014 Feb 10:9:20.
doi: 10.1186/1750-1172-9-20.

Accelerating development, registration and access to medicines for rare diseases in the European Union through adaptive approaches: features and perspectives

David Uguen  1 , Thomas Lönngren, Yann Le Cam, Sarah Garner, Emmanuelle Voisin, Carlo Incerti, Marc Dunoyer, Moncef Slaoui
Affiliations

Accelerating development, registration and access to medicines for rare diseases in the European Union through adaptive approaches: features and perspectives

David Uguen et al. Orphanet J Rare Dis. .

Abstract

There is growing recognition that the current research-and-development (R&D) and innovation-regulation ecosystem could be made more efficient to stimulate and support access to innovative therapies for those patients with rare, life-threatening diseases for which there are no adequate licensed therapies. New and progressive thinking on the principles and processes of drug development and regulation are needed in rare disease settings in order to ensure developments are financially sustainable. This paper presents perspectives on the current and emerging schemes for accelerating development of and access to medicines for rare diseases in the European Union.

PubMed Disclaimer

References

    1. Rollet P, Lemoine A, Dunoyer M. Sustainable rare diseases business and drug access: no time for misconceptions. Orphanet J Rare Dis. 2013;8:109. doi: 10.1186/1750-1172-8-109. - DOI - PMC - PubMed
    1. Dunoyer M. Accelerating access to treatments for rare diseases. Nat Rev Drug Discov. 2011;10:475–6. - PubMed
    1. Eichler HG, Oye K, Baird LG. et al.Adaptive licensing: taking the next step in the evolution of drug approval. Clin Pharmacol Ther. 2012;91:426–37. doi: 10.1038/clpt.2011.345. - DOI - PubMed
    1. European Medicines Agency. Roadmap to 2015. http://www.ema.europa.eu/docs/en_GB/document_library/Report/2011/01/WC50... [Accessed 7 March 2013]
    1. Centre for the Advancement of Sustainable Medical Innovation. Adaptive Licensing. http://casmi.org.uk/adaptive-licensing/ [Accessed 7 March 2013]

Publication types