EU Changes Required to Encourage Childhood Cancer Drug Research

European Union exemptions may not facilitate research into the use of new drugs for paediatric cancers

medwireNews: More should be done to encourage oncology drug manufacturers to investigate the use of their agents in children with cancer, say UK and French organisations.

Changes in the European Union (EU)’s Paediatric Regulation legislation could avoid unnecessary delays to the development of potential paediatric oncology drugs, suggest the Institute of Cancer Research (ICR) in London, UK, and the European Consortium for Innovative Therapies for Children with Cancer in France.

Currently, pharmacology companies can apply for waivers for new drugs from being tested in patients younger than 18 years old, even when there is reason to believe the agent may be effective for paediatric cancers.

The institutes released data showing that 27 of the 28 oncology agents approved by the European Medicines Agency between 2007 and 2012 had a mechanism of action with potential to treat paediatric tumours.

However, 14 of the agents were given a waiver allowing them exemption from a Paediatric Investigation Plan (PIP) because the specific adult condition the drug is licensed for does not occur in children, even though the agents may target characteristics found in childhood malignancies, such as ALK or EGFR Mutations.

"Modern cancer treatments are often targeted at genetic features of the tumour that may be common to a number of tumour types, and to adults' and children's cancers,” explained Professor Alan Ashworth, Chief Executive of the ICR, in a press release.

“That means a drug developed for a cancer in adults could also be effective against a cancer affecting a completely different part of the body in children. The way EU rules are implemented fails to take this into account.”

PIPs were submitted for eight drugs over the study period, while two drugs were not considered suitable for PIPs. Just four drugs were approved for paediatric tumours.

The alternative “orphan drug” route for approving cancer drugs for paediatric patients was also considered ineffective by the institutions, with none of the 25 orphan medicines approved by the European Medicines Agency later registered as a treatment for a childhood malignancy.

“Speeding up innovation is a major goal for the European paediatric oncology community,” commented Gilles Vassal, Head of Clinical Research at Gustave Roussy and Chair of the European Consortium for Innovative Therapies for Children with Cancer, in a press release.

“Setting up cooperation between academia, regulatory bodies, industry and parent organisations is paramount and will be a key success factor.”

Source of information

Institute of Cancer Research, UK, press release

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