Call for European Union Regulation on Paediatric Medicine to be changed
Potentially life-saving drugs are being denied to children with cancer, because they are not being put through the necessary clinical trials, say scientists who are calling for European Union rules to be changed.
The Institute of Cancer Research (ICR), based in London, UK says the current European Union Regulation on Paediatric Medicine allows pharmaceutical companies to opt out of children's trials if a new drug intended for an adult cancer does not occur in children, even where there is evidence that the drug could be effective in children.
These so-called ‘class waivers’ allow pharma companies to avoid the implementation of Paediatric Investigation Plans (PIPs), which are supposed to give companies an incentive to test their products in children by offering longer market exclusivity.
Although waivers are appropriate when an adult cancer drug will not work in childhood cancers, they are often granted even when evidence shows that a drug for adult cancers has a mechanism of action that could in addition treat childhood cancers.
The ICR is therefore calling for urgent modifications to the rules to make sure pharma companies test more of their drugs in children.
The current system is failing to provide children with access to new treatments that could add years to their lives
The organisation is pushing for a rule change in collaboration with the European Consortium for Innovative Therapies for Children with Cancer (ITCC), based in France, after research found that of 28 cancer drugs approved for adult marketing authorisation in Europe since 2007, 26 work in a way that is relevant for childhood cancers.
Yet 14 have been exempted from being tested in children because the specific adult condition for which the drug is developed does not occur in children.
Drugs have been approved for treating adult cancers with mutations in the ALK or EGFR genes, for example, but the manufacturers have been granted waivers from testing them in children, even though ALK and EGFR mutations have been shown to play a role in some childhood cancers.
The research by the ICR and the ITCC also showed that the European Commission’s alternative route for getting cancer drugs to children – its regulation on rare, or ‘orphan’, conditions – is not effective. Of the 25 EU-approved orphan medicinal products for cancer, none was registered for children in a different cancer type from that in adults.
The ICR says there should be stronger financial incentives to compensate pharmaceutical companies for the financial challenge of developing drugs for small patient populations. Some pharmaceutical companies already voluntarily submit PIPs based on mechanism of drug action, but others do not.
Professor Alan Ashworth, Chief Executive of the ICR, said: 'It’s essential that ground-breaking cancer treatments are tested not only in adults but also in children, whenever the mechanism of action of the drug suggests they could be effective. That requires a change to EU rules, since the current system is failing to provide children with access to new treatments that could add years to their lives.
'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. 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.'
Professor Gilles Vassal, Head of Clinical Research at Gustave Roussy and Chair of the European Consortium for Innovative Therapies for Children with Cancer, added: 'The European Paediatric Medicine regulation significantly changed the landscape of drug development in children. However, there is an urgent need to change its implementation in order to meet the need for new innovative medicines to cure children and adolescents suffering life-threatening malignancies.
'Speeding up innovation is a major goal for the European paediatric oncology community. Setting up co-operation between academia, regulatory bodies, industry and parent organisations is paramount and will be a key success factor.'