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WP5: Validation of new methods within clinical as well as regulatory settings

In WP5, a comparison between conventional and new methods will be provided, which is a final applicability step in which current knowledge is lacking and is needed to fill the gap between theoretical development and real life implementation. As such, it will help to understand strengths and weaknesses of new methods and to normalise their acceptance in rare diseases.

Furthermore, an innovative taxonomy-based structure of ASTERIX results will be issued to ensure their generalisation to types of diseases, rather than to single disease models, thereby widening and easing their potential application.

Finally, testing acceptability of methods with both regulators and patients will keep a focus on end-users, ensuring relevance and acceptability of recommendations to facilitate their uptake in routine practice.

As a result, the researchers of WP5 will provide:

  • New classification reference linking disease characteristics with methodologies;
  • Framework for clinical trials in small populations integrating new and improved methodology including recommendations for use based on the classification reference;
  • Guidance for implementation of this framework for clinical trials in small populations.


Leader WP5: Dr. Ferran Torres, Universitat Autònoma de Barcelona


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