Successful private-public funding of paediatric medicines research: lessons from the EU programme to fund research into off-patent medicines

Ruggieri L, Giannuzzi V, Baiardi P, Bonifazi F, Davies EH, Giaquinto C, Bonifazi D, Felisi M, Chiron C, Pressler R, Rabe H, Whitaker MJ, Neubert A, Jacqz-Aigrain E, Eichler I, Turner MA, Ceci A (2015)

Publication Type: Journal article

Publication year: 2015

Journal

European Journal of Pediatrics Springer Verlag (Germany)

Book Volume: 174

Pages Range: 481-91

Journal Issue: 4

DOI: 10.1007/s00431-014-2398-z

Abstract

The European Paediatric Regulation mandated the European Commission to fund research on off-patent medicines with demonstrated therapeutic interest for children. Responding to this mandate, five FP7 project calls were launched and 20 projects were granted. This paper aims to detail the funded projects and their preliminary results. Publicly available sources have been consulted and a descriptive analysis has been performed. Twenty Research Consortia including 246 partners in 29 European and non-European countries were created (involving 129 universities or public-funded research organisations, 51 private companies with 40 SMEs, 7 patient associations). The funded projects investigate 24 medicines, covering 10 therapeutic areas in all paediatric age groups. In response to the Paediatric Regulation and to apply for a Paediatric Use Marketing Authorisation, 15 Paediatric Investigation Plans have been granted by the EMA-Paediatric Committee, including 71 studies of whom 29 paediatric clinical trials, leading to a total of 7,300 children to be recruited in more than 380 investigational centres.Notwithstanding the EU contribution for each study is lower than similar publicly funded projects, and also considering the complexity of paediatric research, these projects are performing high-quality research and are progressing towards the increase of new paediatric medicines on the market. Private-public partnerships have been effectively implemented, providing a good example for future collaborative actions. Since these projects cover a limited number of off-patent drugs and many unmet therapeutic needs in paediatrics remain, it is crucial foreseeing new similar initiatives in forthcoming European funding programmes.

Authors with CRIS profile

Antje Neubert Department of Paediatrics and Adolescent Medicine

Involved external institutions

National Institute for Health and Medical Research / Institut national de la santé et de la recherche médicale (INSERM) FR France (FR) Gianni Benzi Onlus Pharmacological Research Foundation IT Italy (IT) The University of Liverpool GB United Kingdom (GB) European Medicines Agency GB United Kingdom (GB) University of Sheffield GB United Kingdom (GB) University of Sussex GB United Kingdom (GB) Great Ormond Street Hospital (GOSH) GB United Kingdom (GB) Consorzio per Valutazioni Biologiche e Farmacologiche (CVBF) IT Italy (IT) PENTA Foundation IT Italy (IT)

How to cite

APA:

Ruggieri, L., Giannuzzi, V., Baiardi, P., Bonifazi, F., Davies, E.H., Giaquinto, C.,... Ceci, A. (2015). Successful private-public funding of paediatric medicines research: lessons from the EU programme to fund research into off-patent medicines. European Journal of Pediatrics, 174(4), 481-91. https://doi.org/10.1007/s00431-014-2398-z

MLA:

Ruggieri, L., et al. "Successful private-public funding of paediatric medicines research: lessons from the EU programme to fund research into off-patent medicines." European Journal of Pediatrics 174.4 (2015): 481-91.

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