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

Orphanet Journal of Rare Diseases

Moncef Slaoui⁶  Marc Dunoyer⁶  Carlo Incerti⁴  Emmanuelle Voisin²  Sarah Garner³  Yann Le Cam⁵  Thomas Lönngren¹  David Uguen² 

[1] NDA Advisory Services Ltd, Leatherhead, Surrey, UK;Voisin Consulting Life Sciences, Paris, France;Centre for the Advancement of Sustainable Medical Innovation, London, UK;Genzyme Corporation, Cambridge, USA;European Organisation for Rare Diseases (EURORDIS), Paris, France;GSK London, London, UK

关键词: Patient access;    Adaptive licensing;    Rare diseases;    Orphan medicinal products;   

Others  :  863256
DOI  :  10.1186/1750-1172-9-20

 received in 2013-07-11, accepted in 2014-02-07,  发布年份 2014

【 摘 要 】

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.

【 授权许可】

   
2014 Uguen et al.; licensee BioMed Central Ltd.

【 参考文献 】

• [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. BioMed Central Full Text
• [2]Dunoyer M: Accelerating access to treatments for rare diseases. Nat Rev Drug Discov 2011, 10:475-6.
• [3]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.
• [4]European Medicines Agency: Roadmap to 2015. http://www.ema.europa.eu/docs/en_GB/document_library/Report/2011/01/WC500101373.pdf webcite [Accessed 7 March 2013]
• [5]Centre for the Advancement of Sustainable Medical Innovation: Adaptive Licensing. http://casmi.org.uk/adaptive-licensing/ webcite [Accessed 7 March 2013]