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EU Clinical trial regulation in the environment of rare diseases: time for a change

The clinical trial directive 2001/20/EC is out for public consultation. This session will draw the attention of the challenges faced by Small and Medium Enterprises (SME) companies when developing new products for the rare diseases that affect a very limited number of patients and discuss possible options to overcome the challenges.

Indeed, with only a few patients in each country, clinical trials need to be conducted in many countries in order to enrol the number of patients required to demonstrate benefit/risk. The management of these clinical trials is associated with several issues that slow down the overall process of drug development: administrative hurdles associated with non harmonised regulatory authorisation process as well as cost pressure linked to the need to outsource local activities to several consultants to prepare and manage submission to health authorities and ethic committees.

Today, Europe must streamline its system and all stakeholders should raise their voice to propose specific approaches in order to facilitate the development of new drugs for orphan drugs in a timely manner.

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Correspondence to Martine Zimmermann.

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Open Access This article is published under license to BioMed Central Ltd. This is an Open Access article is distributed under the terms of the Creative Commons Attribution 2.0 International License (, which permits unrestricted use, distribution, and reproduction in any medium, provided the original work is properly cited.

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Zimmermann, M. EU Clinical trial regulation in the environment of rare diseases: time for a change. Orphanet J Rare Dis 5 (Suppl 1), P19 (2010).

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