The manuscript “Ethical and procedural issues for applying researcher-driven multi-national paediatric clinical trials in and outside the European Union: the challenging experience of the DEEP project” has been published on the BMC Medical Ethics journal.
Viviana Giannuzzi, researcher from Fondazione Benzi coordinated this work that aims to evaluate the impact of the different local and national rules and procedures and of their complexity on the paediatric trial authorisation process in different European and non-European countries from the preparation of submission package to the release of authorisation and approval.
Trials involving “small populations”, such as children and patients affected by rare diseases, are typically multi-centre and multi-national. For this type of studies, the preparation of the Clinical Trial Application can be very challenging. Besides Good Clinical Practice guidelines, different rules and procedures set out at a local level need to be complied with.
This challenge has been tackled within the DEEP - DEferiprone Evaluation in Paediatrics project (FP7 HEALTH-F4-2010-261483), aimed at studying Deferiprone in children. In the framework of this project, the paediatric multi-national trial DEEP-2 (EudraCT 2012–000353-31; NCT01825512) involved paediatric patients from 1 month to less than 18 years affected by transfusion-dependent haemoglobinopathies.
Based on the experience from DEEP-2 trial, the article highlights the differences among procedures and requirements for the application of a paediatric trial. In the European Union, the timeframes for submission are generally harmonised but longer. In non-European countries, delays are caused by national dispositions, but the entire authorisation process resulted faster with less requests from Ethics Committees or Competent Authorities.
Authors have also highlighted that the upcoming application of Regulation (EU) 536/2014 is expected to harmonise practices in Europe and possibly outside. Networks on paediatric research acting at international level will be crucial in this effort!
Discover more by reading the full article here!