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Extrapolation or controlled trials in paediatrics: the current dilemma

26 Aug 2017

Historically, it has been difficult to conduct controlled studies in children because of the paucity of large collaborative research networks, scarce availability of funding and lack of interest from pharmaceutical companies. Thanks to the Best Pharmaceuticals for Children Act in USA and to a dedicated legislation for paediatric medicines development (Paediatric Regulation) in the European Union, this dire situation is very much improved.1 2

Undoubtedly, in some paediatric specialities, such as paediatric rheumatology, this legislation has been a great success.3 At least to some extent, this success seems attributable to the existence of two large networks, namely the Pediatric Rheumatology Collaborative Study Group (PRCSG) (, covering North America, and the Paediatric Rheumatology International Trials Organisation (PRINTO) (, covering more than 60 countries worldwide.4 Through coordinated and collaborative activities over the preceding two decades, these two networks have effectively worked in academia (over...

Click here to view the full article which appeared in Archives of Disease in Childhood