Paediatric Medicines

For Advocacy on the Paediatric Medicines Regulation 

For SIOPE publications on paediatric medicines

Anticancer therapies for children and adolescents

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.

Since few paediatric cancer studies have been performed for licensing purposes within the last 30 years, 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 was 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 be able to access the same innovative anticancer therapies developed for the treatment of adult cancers.

EU Paediatric Regulation

The EU Paediatric Regulation 1901/2006 was expected to significantly increase the number of those drugs in clinical development for children and adolescents in Europe. However, the Regulation didn’t truly address the needs of young people with cancer, as the number of new oncology drugs in paediatric development remains low in Europe (still a 10-fold difference if compared to the US in the number of new anticancer drugs available for clinical research).

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:

  • a) facilitating the development and availability of medicines for children aged 0 to 17 years;
  • b) ensuring that medicines for use in children are of high quality, ethically researched and authorised appropriately; and
  • c) improving the availability of information on the use of medicines for children.

The Regulation would also like to avoid to subject children to unnecessary trials and to delay the authorisation of medicines for use in adults.


Co-organised by SIOPE and ITCC, the ACCELERATE Platform was created after the 2013 ACCELERATE Paediatric Oncology Conference to intensify cooperation between all stakeholders – from academia, parents, regulatory agencies and industry – and to make proposals that address the identified bottlenecks.  At every annual ACCELERATE Paediatric Oncology Conference, the working groups report on their advances and their proposals are discussed with all participants.

As the European Commission prepared to launch a consultation on the impact of the EU Regulation, members of the ACCELERATE platform – including the Unite2Cure network of parents, parent organisations and patient advocates – proposed a few amendments:

  • Ensure that the obligation to undertake a Paediatric Investigation Plan (PIP) is based on how a drug works and its capacity to address an unmet medical need in children, rather than the type of disease in adults for which it is first introduced;
  • Set up a mechanism to choose the best potential drugs and prioritise, among drugs developed by different companies, in relation to the real needs of children affected by rare cancers;
  • Reduce delays in paediatric medicines reaching children by enabling Paediatric Investigation Plans (PIPs) to be submitted not later than the start of pivotal trials in adults, if paediatric biological, preclinical and preliminary clinical data are available to better evaluate the potential therapeutic benefit in the paediatric population;
  • Add provisions for more effective and flexible rewards for companies undertaking early and timely Paediatric Investigation Plans and those researching therapies specifically for cancers which only occur in children.

Although the Paediatric Regulation did change the landscape of drug development before 2007, it did not represent a solution for paediatric cancers.  Too few drugs targeting paediatric oncology are currently under development, in spite of cancer remaining the first cause of children’s death by disease in Europe. These proposals will be discussed at the high-level ACCELERATE Paediatric Oncology Conference (the 2020 edition will take place on 6-7 February in Brussels, Belgium).

More information: