European Cancer Community Welcomes European Parliament Support for Children with Cancer
Safe and effective anticancer therapies
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 SIOPE and ITCC, 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 (the 2020 edition will take place on 6-7 February in Brussels, Belgium).
In June 2018, the ACCELERATE platform was reorganised to strengthen international cooperation in order to improve global development of new paediatric oncology drugs.
Members of the ACCELERATE Platform – including the Unite2Cure network of parents, parent organisations and patient advocates – 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.
- ACCELERATE Platform
- Press Release: SIOPE and Unite2Cure react to the Paediatric Regulation report
- For Research on paediatric medicines
- SIOPE publications on the Paediatric Regulation