The aim of the EU Pharmaceutical Strategy is to ensure Europe’s supply of safe and affordable medicines to meet patients’ needs and support the European pharmaceutical industry’s innovation efforts.
Our paediatric oncology community is delighted that the Strategy refers to the evaluation of the Paediatric and Orphan Regulation and mentions a plan on drug shortages. The new EU Pharmaceutical Strategy is an opportunity to make lifesaving differences for young cancer patients by addressing its key pharmaceutical challenges. These include: The poor progress made regarding high-risk paediatric tumours due to lack of access to targeted innovation; major inequalities in access to essential medicines across Europe; and inadequate access to innovative therapies for children and adolescents with cancer.
It remains an inescapable fact that cancer continues to be Europe’s leading cause of death by disease for children aged over one. Each year, more than 35,000 children and young people are diagnosed with cancer and more than 6,000 die of cancer. These grim figures highlight Europe’s need to recognise that paediatric cancer remains an urgent health and socio-economic issue. Medicines have played a crucial role in improving the quality and length of life for childhood cancer patients, but progress has plateaued with the currently available choice of therapies. Although adult cancer therapies are evolving rapidly, with more innovative medicines reaching Europe, childhood cancer patients regrettably do not benefit. The Paediatric Regulation was expected to be more relevant to the development of new medicines for children, but after 10 years of implementation it has left paediatric cancer needs wanting.
“Only nine anti-cancer medicines were authorised for a specific paediatric indication since 2007, in contrast to over 150 for adult cancers. This slow pace of innovation cannot serve paediatric cancer patients. Progress requires solid support for dedicated international academic research platforms, crosslinked to and informed by adult cancer and industry-driven research, and regulatory changes.”
Despite their high collective burden, the rarity of individual paediatric cancers has translated into very limited market-driven innovation. Since 2007, only nine anti-cancer medicines were authorised for a specific paediatric indication, in contrast to over 150 for adult cancers. This slow pace of innovation cannot serve paediatric cancer patients appropriately. Progress requires solid support for dedicated international academic research platforms, crosslinked to and informed by adult cancer and industry-driven research and regulatory changes.
ACCELERATE (www.accelerate-platform.org) is a multi-stakeholder platform formulating evidence-based recommendations to address the challenges of paediatric oncology medicine development. Created in 2015, it is a partnership between academia, industry, regulatory agencies (EMA and FDA) and patient advocate groups that aims to improve and speed up the development of innovative therapies. Specifically, this unique initiative aims to:
- Facilitate prioritisation of assets through science to address unmet medical needs;
- Strengthen international cooperation for global developments;
- Increase access to innovation for children and adolescents;
- Anticipate long-term follow up for young patients exposed to new anti-cancer medicines;
- Contribute to improving the regulatory environment.
ACCELERATE has already delivered successfully on this agenda and is eager to contribute to the design of the EU Pharmaceutical Strategy and to its successful implementation.
We welcome the European Commission’s recognition of the need to evaluate the regulatory framework and its implementation more closely. However, the needs of children and adolescents with cancer must be fully and urgently considered. SIOP Europe has implemented a comprehensive stakeholder mobilisation effort to ensure that paediatric cancer community views are well represented as the new EU Pharmaceutical Strategy evolves. The principal asks are:
- Improved access to medicines for children and adolescents with cancer – including in relation to innovative therapies, countering shortages, and advancing affordable prices of new expensive medicines.
- Support for a legislation that enables faster and more efficient development of innovative medicines for children and adolescents with cancer, including incentives for the development of anticancer drugs targeting alterations specific to paediatric malignancies and for drug repurposing.
The EU Pharmaceutical Strategy is a unique opportunity to address these major unmet needs in paediatric cancer and we urge the Commission to seize this chance to make a pivotal change for our youngest citizens.
For more information, visit SIOP Europe: www.siope.eu
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