Developments in the EU Paediatric Regulation


Innovative Medicines

3. Fig 1Thanks to the increasing introduction of molecular targeting and personalised medicine strategies, innovative medicines and treatments are nowadays available for adults with cancer. Widespread access to these therapies could also treat children and adolescents diagnosed with this disease, and even reduce the long-term side effects of the current treatments. However, despite these therapies’ potential to target paediatric malignancies, innovation in the research and development of new cancer medicines for children and adolescents has not been sufficient and, today, less than 1 in 10 children in relapse with a terminal cancer have access to these therapies. At the same time, cancer is still the leading cause for death by disease among children, with 6,000 young people dying of cancer every year in Europe. Why children cannot access innovative therapies?

Developing New Childhood Cancer Therapies

Paediatric oncology drug development in Europe is currently regulated by the EU Regulation on Paediatric Medicines (EC No 1901/2006), and in 2017 it will be 10 years since its entry into force. Although the Regulation helped in speeding up paediatric drug development, the European Society for Paediatric Oncology (SIOPE) observed that medicines, options and investments to cure childhood cancers have not been sufficient so far. During these years, pharmaceutical companies managed to avoid the obligation to investigate and develop drugs for the paediatric population, and oncology drug development was mainly driven by the adult cancer “market”. More specifically, companies often obtained waivers from the obligation to investigate the potential benefit of a drug in a child, if the cancer for which the drug was originally developed in adults does not exist in children. However, such drugs can still be used to treat other childhood cancer types (for example, a new drug for adult lung cancer can benefit children with neuroblastoma).

The Added Value of ACCELERATE

Significant changes and meaningful incentives are therefore urgently needed to give children and adolescents suffering from this disease better chances to survive and thrive. Interestingly, the past ten years were also marked by a close interaction between all the stakeholders who wanted to improve the Regulation in this direction: academic researchers and healthcare professionals; pharmaceutical representatives; officers from the drug regulatory agencies in Europe; and patient/parent advocates. In December 2013 SIOPE, ‘Innovative Therapies for Children with Cancer (ITCC) and ‘Cancer Drug Development Forum’ (CDDF) launched the multi-stakeholder platform ‘ACCELERATE - Innovation for Children with Cancer’ to facilitate this sustained interaction and support the stakeholders’ joint efforts to analyse the current situation, identify bottlenecks and design solutions to improve new oncology drug development for children and adolescents.

European Parliament Resolution

3. LatestAfter an intensive advocacy campaign, on 15th December 2016 SIOPE together with the ACCELERATE partners and Cancer Research UK welcomed the adoption of a new European Parliament Resolution seeking to improve the current EU Regulation on Paediatric Medicines. This Resolution aims to limit pharmaceutical companies’ waivers to the obligation to investigate drugs for children and calls upon the European Commission to modernise the current legislation and facilitate a rapid and better prioritised development of innovative therapies, ensuring that it goes as far as it can for children with cancer across Europe.

Learn More at the ACCELERATE Event (2-3 March 2017)

3. Fig 3All these recent developments as well as the next steps to be taken will be discussed at the upcoming ACCELERATE Paediatric Oncology Conference, which wil l take place on 2-3 March 2017 in Brussels. Formerly known as the CDDF-ITCC-SIOPE Paediatric Conference, this conference represents the occasion for high-level experts to reflect upon successes and failures in paediatric development, share new initiatives in clinical development, discuss proposals on how to accelerate paediatric oncology drug development, and comprehensively review the 10 years’ report on the EU Paediatric Regulation (for more information, please see here). Everyone with a sheer interest in this topic is very welcome to attend this event, which will potentially make a breakthrough in public awareness on this important topic.