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

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.Conclusion: 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.

A Literature Review of the Pharmacokinetics and Pharmacodynamics of Dobutamine in Neonates

“…Changes to European drug legislation have meant that the European Union has been able to sponsor ongoing drug trials such as the NEOCIRC project funded by the Seventh Framework Programme [58]. This hopefully will help to answer some of the queries highlighted in this review and will enable a larger multicenter trial to be performed, which may provide results that can alter clinical practice and regulate the use of dobutamine.…”

“…The European Commission (EC) recently established a regulation, which encourages research into the use of offlabel medications in the pediatric population [54,58]. Both the European Medicines Agency in their priority list for studies into off-patent pediatric medicinal products and the US National Institutes of Health in their Best Pharmaceuticals for Children Act have highlighted dobutamine as a drug that requires further investigation [18,35].…”

Paediatric Drug Development and Formulation Design—a European Perspective

“…Acknowledging that information on the pharmaceutical design of paediatric drugs in relation to patient outcomes was scarce and fragmented, the Paediatric Regulation has triggered a variety of research opportunities (12,13). In addition, several research consortia were established, e.g.…”

Quality Assessment in the Extemporaneous Pediatric Preparation of Powder Papers Containing Crushed Tablets of Spironolactone or Amiodarone

“…[22] Another issue to consider is the few clinical researches in this vulnerable population as there are difficulties of many types. [5] Regulatory authorities have issued guidelines [9,[22][23][24][25][26] and in several countries incentives have been granted for their development. [27,28] However, these incentives are not at all attractive to pharmaceutical laboratories because pediatric medicines have certain disadvantages: they are considered therapeutic orphans, prescribed in rare diseases and in a limited number of patients.…”