Europe must do more to ensure children don’t miss out on life-saving treatments
The EU Paediatric Medicines Regulation has resulted in far fewer treatments being made available for children than there should be. Glenis Willmott MEP reviews the regulation and points out what crucially needs changing regarding current EU-wide policies on prevention, education, screening, treatment, care or research relating to paediatric cancer
Cancer remains the single biggest cause of death by disease for children in Europe, with 35,000 children and adolescents diagnosed every year. While survival rates have certainly improved, 6,000 young people will die from cancer each year.
As with other rare diseases, research into paediatric cancer is often not seen as a priority because the pool of patients is relatively small and the profit margins low; so despite the progress that has been made in treating cancer, very few innovative treatments have been developed specifically for childhood cancer. Those treatments that are available cause severe side-effects in two thirds of paediatric cancer survivors.
The European Union’s Paediatric Regulation was intended to address the lack of treatments for paediatric conditions by requiring developers to test all new medicines for their potential to treat children. The Regulation has certainly had some success: since 2007, more than 800 Paediatric Investigation Plans have been agreed, with 41 medicines developed with a specific paediatric indication and a further 120 where paediatric dosing has been improved.
However, for oncology the impact of the regulation has been disappointing, with only two innovative targeted anti-cancer drugs authorised for paediatric indications. There are a number of reasons for this but one of the main problems is that under the legislation, waivers are granted for medicines that are intended to treat a condition that does not occur in children. Most adult cancers do not occur in children and vice versa, and so many new oncology drugs are not investigated for their potential to treat paediatric cancers.
The problem with this is that, even where a type of cancer only occurs in adults, the mechanism of action of a drug may still be effective in treating a paediatric cancer. For example Crizotinib is used in the treatment of ALK+ lung cancer, which does not occur in children. The same molecular abnormalities that cause this type of cancer have been observed in a number of paediatric cancers, such as lymphoma and sarcoma. However, because the drug was developed for a condition that does not occur in children, the developers received a paediatric investigation waiver and children in Europe were denied access to this potentially life-saving medicine.
Researchers estimate that in the first five years after the Paediatric Regulation came into force, 26 new drugs were developed for adult cancer that had potential to treat paediatric conditions, but over half received a waiver.
The Commission is due to report on the success of the Paediatric Regulation in 2017 and may consider revising it. I’ve been working with Unite2Cure, which represents the parents of children with cancer, calling for the Commission to amend the legislation so that the decision to grant a waiver is not based purely on the type of condition, but also takes into account the biology of the cancer and the drug’s mechanism of action.
There are other problems we’d like to see addressed too, such as the fact that Paediatric Investigation Plans (PIPs) for new anti-cancer drugs are often started late in a drug’s development as the investigators wait for it to show promise in adults first, meaning that some children may miss out on potentially life-saving treatments. Similarly, there’s nothing to stop investigators ending a promising paediatric trial early if they’re not getting the results they want in an adult trial.
When it comes to clinical trials, cross border trials are particular important for childhood cancer, as there often aren’t enough patients in one country to make a trial viable; for many children with cancer, a clinical trial may be their only chance of survival. Another important piece of EU legislation in this regard is the new Clinical Trials Regulation, which will make it much easier to carry out cross-border trials.
I was interested to read about recent research into a potential treatment for malignant rhabdoid tumours, a devastating form of cancer for which there is currently no cure.
Scientists at the Institute for Cancer Research found that using existing medicines to inhibit two types of protein effectively killed the cancer cells. This is promising research and the researchers are hopeful it may be relevant to other types of cancer too. Nevertheless, they also highlighted that it is currently too easy for pharmaceutical companies to avoid evaluating their drugs in children.
So while progress is clearly being made in Paediatric Oncology, there’s more that Europe could do to facilitate it and I hope the European Commission will listen to some of these recommendations.
The UK organisation Christopher’s Smile, who raise money for research into paediatric cancer and advocate for better policies in this area, have a vision for “every child diagnosed with childhood cancer to not only survive, but to reach adulthood enjoying a good quality of life.” I believe that should be the European Union’s aim too.