Children with cancer can’t wait any longer
The EU Paediatric Medicines Regulation has benefited a series of childhood diseases, yet only two innovative anti-cancer drugs have been authorised so far. Francoise Grossetete MEP analyses the impact of the legislation and points out what more needs to be done to offer quick and tangible results to children with cancer
The Paediatric Regulation came into force in the European Union on 26 January 2007. In early 2017, the European Commission – the EU body that drafts and updates new laws – will decide whether to revise the law or leave it as it stands. This amending regulation was deemed necessary because the legislative framework at the time had failed to address the lack of new paediatric medicines being put on the market and the lack of innovation thereof. It had a strong impact on development of paediatric medicines, partly because it obliged companies to conduct a Paediatric Investigation Plan (PIP) for each new molecule they wanted to market. In this regard, it helped improve the situation and led to tangible benefits for a series of childhood diseases.
However, we now clearly see that there is still a long way forward, especially in certain therapeutic areas such as paediatric oncology. Childhood cancer remains the first cause of death by disease in children aged one year and over, and 6,000 young people die of cancer each year in Europe. Those who are treated often suffer from treatment-related side effects. Yet, only two innovative targeted anti-cancer drugs were authorised for a paediatric malignancy since the Paediatric Medicines Regulation came into force. We clearly see that something urgently needs to be done.
According to my shared understanding, the obstacles are threefold. First, the legal requirement to pursue a PIP is too often waived, because the drug is being developed for an indication that does not occur in children. Second, the PIPs, conducted at a very early stage in the process, are often complex and inflexible, which leads companies trying to bypass them or to simply abandon them later in the process. Third, the current system of rewards and sanctions is not appropriate and effective enough, leaving pharmaceutical companies with little incentive to invest in a field where return on investments are far from guaranteed.
For all those reasons, I believe that the current regulation must be adapted if it is to deliver concrete results for children with cancer. A full legislative review may not be needed, as it could take time and seriously put at risk the basis of the current legislation, which I think has proved successful even if we now see its limits. Instead, I think changes could be introduced through regulatory procedures, guidelines from the EMA for example. To work in this direction and ahead of the publication of the European Commission’s implementation report on the Paediatric Medicines Regulation next year, the European Parliament has decided to adopt a resolution to highlight its priorities.
The final text is yet to be adopted but as it stands, it tries to address the main shortcomings I have mentioned above. It calls to take into consideration not only cancer-type-based paediatric development plans, but also plans based on the mechanism of action matching the tumour’s biology. It asks companies to start paediatric development as soon as possible, in order to enable early regulatory dialogue and joint development with the European Medicines Agency, allowing for more feasible PIP.
Finally, it requires the European Commission to assess how different types of rewards could be best utilised to drive and accelerate clinical development of drugs for childhood disease, and specifically for those cancers which occur only in children.
This resolution is obviously only a first step. I have high expectations for the Commission’s report due to be published in 2017, which should make a good assessment of the situation and of the existing margins of manoeuvre for adapting the current legislative framework. Whatever is the chosen solution in the end, it will have to be able to quickly deliver tangible results.