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Until a few years ago, a1 majority of pharmaceutical drugs did not have any label for paediatric use; this situation prompted both the USA and European Union (EU) to pass specific legislation for clinical trials in children.2,–,4 In Europe, extensive efforts have been made to facilitate drug development for children by setting up the Paediatric Committee at the European Medicines Agency (EMA), by implementing particular rules to enhance paediatric drug development, including orphan drugs which are often for children, and more recently by establishing the European Network of Paediatric Research at the EMA.5,–,7 In the light of these efforts it is essential that other elements of the regulatory system work in the same direction and are not counterproductive. In a case study, we wish to highlight hurdles related to the ethical assessment that need to be addressed.
The EUROFEVER project and PRINTO network: a case study
Assessment of drug effectiveness requires collaborative data gathering by several clinical and academic centres. For rare diseases and for paediatric studies in particular, this network must be widespread comprising many centres on a global level. As a result of the Swedish EU Presidency Conference ‘Assessing Drug Effectiveness’ on 29 July 2009, an initiative was taken to define factors of success and problems encountered by European clinical projects and networks from a drug effectiveness perspective. One of these projects was the EUROFEVER registry, aimed at collecting information on the clinical presentation, outcome and response to treatment of patients affected by autoinflammatory diseases in childhood.4 8
Autoinflammatory diseases are a group of rare conditions secondary to mutations in …
Funding The Swedish Medical Products Agency, The IMI project BT-CURE (Grant agreement No. 115142-1) and the Biobanking and Molecular Resource Infrastructure of Sweden (BBMRI.se).
Competing interests None.
Provenance and peer review Not commissioned; externally peer reviewed.
Data sharing statement Data of this study are open for access.