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A European Network of Paediatric Research at the European Medicines Agency (Enpr-EMA)
  1. Nicolino Ruperto1,
  2. Irmgard Eichler2,
  3. Ralf Herold2,
  4. Gilles Vassal3,
  5. Carlo Giaquinto4,
  6. Lars Hjorth5,
  7. Adolf Valls-i-Soler6,
  8. Christina Peters7,
  9. Peter J Helms8,
  10. Agnès Saint Raymond2
  1. 1Pediatric Rheumatology International Trials Organisation (PRINTO) at IRCCS G Gaslini, Pediatria II, Reumatologia, Genova, Italy
  2. 2European Medicine Agency and Enpr-EMA, London, UK
  3. 3European Consortium for Innovative Therapies for Children with Cancer (ITCC) at Institut Gustave Roussy, Villejuif, France
  4. 4Paediatric European Network for Treatment of AIDS (PENTA), ONLUS at Dipartimento di Pediatria, Padova, Italy
  5. 5Pan-European Network for Care of Survivors after Childhood and Adolescent Cancer (PanCare), Consultant Paediatric Oncology & Haematology, Department of Paediatrics Lund, Skåne University Hospital, Clinical Sciences Lund University, Lund, Sweden
  6. 6European Neonatal Network (EuroNeoNet), Neonatal Intensive Care and Clinical Epidemiological Unit, Department of Paediatrics, University of the Basque Country, Bilbao, Spain
  7. 7European Blood and Marrow Transplantation Group (EBMT) at Stammzelltransplantations-Einheit, St Anna Kinderspital, Wien, Austria
  8. 8Scottish Medicines for Children Network at Child Health University of Aberdeen, Aberdeen, UK
  1. Correspondence to Nicolino Ruperto, IRCCS G. Gaslini, Pediatria II – Reumatologia, Pædiatric Rheumatology International Trials Organisation (PRINTO), EULAR Centre of Excellence in Rheumatology 2008-2013, Via G Gaslini, 5, Genova 16147, Italy; nicolaruperto{at}

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Conducting clinical trials in the paediatric population is difficult for a host of reasons that include logistical, methodological, financial and ethical problems. Indeed for many paediatric conditions, their low prevalence means that multicentre studies performed on an international scale often represent the only possibility to gather a sufficient number of patients (ie, to obtain clinically and statistically valid results) over a reasonable period of time, especially for drug trials. However, such studies are difficult to conduct for several reasons including ethical issues such as assignment to placebo, lack of adequate paediatric methods to assess response to therapy, lack of adequate paediatric formulations, the need for specific study designs, inadequate funding as the consequence of the small potential market and limited funding for investigator led academic studies. In addition, there are several bureaucratic constraints related to ethics approval and clinical trial authorisation that often hinder investigator led academic sponsored clinical trials, which do not have the extensive logistical support normally provided by pharmaceutical companies.1 The overall result is that, until recently, evidence regarding the safety and effectiveness of available treatment regimens tended to be from small, open, uncontrolled trials or from anecdotal reports and non-randomised case series.

From a logical and scientific point of view, one of the key issues to overcome these problems is to work with established clinical trials networks that have a wide international representation and a good scientific reputation.

In this regard, the adoption of legislations to encourage paediatric clinical trials both in Europe and in the USA has opened a new era in the …

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  • Competing interests None.

  • Provenance and peer review Not commissioned; externally peer reviewed.