Paediatric Medicines Regulation

Since few paediatric cancer studies have been performed for licensing purposes within the last 30 years, 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. Also known as the ‘Paediatric Regulation’, the EU Regulation on medicinal products for paediatric use 1901/2006 came into force in 2007 to address this issue. Based on rewards, incentives and obligations for pharmaceutical companies, the Regulation aimed to: a) facilitate the development and availability of medicines for children aged 0 to 17 years; b) ensure that medicines for use in children are of high quality, ethically researched and authorised appropriately; and c) improve the availability of information on the use of medicines for children, all this without subjecting children to unnecessary trials or delaying the authorisation of medicines for use in adults.

In the following years, SIOPE was requested to contribute to the revision process of the EMA priority list for studies into off-patent paediatric medicines and to respond to a Consultation on the implementation of the Regulation after 5 years (more information here). Today, as the European Commission prepares to launch a consultation on the social and economic impact of this Regulation , the European paediatric cancer community rings the alarm: less than 1 in 10 children with a terminal cancer have access to potentially life-saving innovative medicines for their disease. Although the Paediatric Regulation did change the landscape of drug development before 2007, it didn’t 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.

This is why SIOPE joined multiple stakeholders from academia, parents, regulatory agencies and industry and created the ACCELERATE platform. Co-organised by CDDF and ITCC, SIOPE, this innovative platforms allows multiple stakeholders to work together, discuss the current hurdles and propose solutions 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. These proposals are then presented at a high-level ACCELERATE Paediatric Conference (whose 2017 edition will take place on 2-3 March 2017 in Brussels, Belgium).

Members of the ACCELERATE platform – including the Unite2Cure network of parents, parent organisations and patient advocates – recently proposed a few amendments to the Paediatric Regulation:

  • Ensure that the obligation to undertake a Paediatric Investigation Plan 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.

In May 2016 a few representatives from the ACCELERATE platform – SIOPE and Unite2Cure – had the opportunity to meet the European Commissioner for Health and Food Safety, Dr.  Vytenis Andriukaitis, and explain the concerns of the paediatric cancer community in this regards. More recently, the participants of a recent European Parliament conference (‘Review of the EU Paediatric Medicines Regulation – Let’s do more for children with cancer’, 7 September 2016, Brussels) welcomed the above ACCELERATE proposals. All these positive developments show how this multi-stakeholder initiative managed to convey the urgent need to speed up innovation also to the European Union at the highest level.

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