Paediatric Medicines Regulation

The need to speed up the development of safe and effective anticancer therapies for children and adolescents as well as the need for more studies to obtain paediatric information for paediatric medicines is today a matter of consensus at the global level.

Within the last 30 years, since few paediatric cancer studies have been performed for licensing purposes, treatment has largely been driven by random phase III clinical trials. These are usually investigator-led in the academic community using off-patent drugs outside of their license indication. Without the help of the pharmaceutical industry, significant progress has been through these studies performed by academic networks; however, there is still a need for a legal obligation for pharmaceutical companies to perform studies to develop medicines for use in the paediatric population, as children in Europe should access the same innovative anticancer therapies developed for the treatment of adult cancers.

Based on this reflection, the EU Regulation on medicinal products for paediatric use came into force in 2007. Also known as the ‘Paediatric Regulation’, the Regulation is based on rewards, incentives and obligations for pharmaceutical companies, in order to accelerate the development of drugs for paediatric diseases with no expected direct return on investment by:

  • Facilitating the development and availability of medicines for children aged 0 to 17 years
  • Ensuring that medicines for use in children are of high quality, ethically researched and authorised appropriately
  • Improving the availability of information on the use of medicines for children


  • Subjecting children to unnecessary trials
  • Delaying the authorisation of medicines for use in adults

The European paediatric oncology community joined its forces in the past years to discuss the current hurdles via an ongoing series of Paediatric Oncology Conferences co-organised by CDDF and ITCC, SIOPE, in association with the ENCCA project. These conferences allowed an exchange platform for all actors – academia, parents and survivors, industry and regulators, policymakers and charities. The same actors decided to create a “Paediatric Platform”, an innovative multi-stakeholder initiative that has established a work programme and concrete action plan to improve the development of new oncology drugs for children and adolescents, to evaluate progress and define further steps to foster more and better cure, including with regards to the paediatric medicines legislation.

More information:


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