Brussels – The Anticancer Fund participated to a public hearing of the European Parliament, focusing on solutions for paediatric and rare cancer.

Lydie Meheus, managing director of the Anticancer Fund, was invited as a speaker to a hearing organised by the Special Committee on Beating Cancer (BECA).

Bartosz ARŁUKOWICZ, the chair of the BECA-committee, started by underlining that childhood cancer remains the principal cause of death by disease in children aged one year and over, and that, with over 100 types of childhood cancer, each case is considered as a rare cancer.

There are more than 5 million rare cancer patients in Europe, which together represent approximately 25% of all cancer cases.

The hearing consisted of two parts: the first part focused on research, treatment and care of paediatric, adolescent and rare cancer, while the second part looked into the rights and needs of rare cancer and paediatric cancer patients, survivors, parents and caregivers.

Rare cancers in need for solutions

Lydie Meheus was the final expert speaking. She cited Mr Mariana Mazzucato, the well-known economist behind the Cancer Mission, who said, “If the goal of Europe is better public health, then Europe could serve its stakeholders by being more entrepreneurial”.

Dr Meheus then proposed several solutions with Europe “in the driver’s seat” to deliver affordable treatments with tangible benefits for patients with rare cancers including children. First, ensure that all marketed drugs with a scientific rationale are available for rare cancers: bring off-patent label drugs on-label - very important for paediatric oncology.

The European Commission STAMP expert group (Safe and Timely Access to Medicines for Patients) works on repurposing off-patent drugs in dialogue with all stakeholders (industry and not for profit). A pilot project from the European Medicines Agency (EMA) on clinical trials, driven by science and patients, is integrated in the new pharmaceutical strategy and foreseen to start in 2021. In this concept, clinical trials are executed with (governmental or philanthropic) public money. The EMA mandate should be expanded to advice on data of off-patent medicines for rare cancers.

We can learn from COVID-19

Dr Meheus also explained that in order to find the best therapy for the patient, independent patient-centred (as opposed to drug-centric) platform trials are needed allowing for the selection of the best treatment. She gave the successful example of the so-called ‘Recovery trial’ for COVID-19 that was based on multi-stakeholder collaboration and data sharing and supported by the UK government and the Bill & Melinda Gates Foundation. It yielded solid evidence both for patented and for generic drugs. The EU should apply the same approach to develop drugs for rare cancers. However, Europe’s Beating Cancer Plan or the Cancer Mission do not foresee funding for this type of non-commercial clinical trials.

Dr Meheus also called for support for public disease registries capturing valuable patient-level data for decision-making and guidelines and added that the Joint Research Centre and the European Health Data Space could play a role to support cancer registries through the Knowledge Centre.

In conclusion, the Anticancer Fund brought a solution to the table for the issue raised that off-label drugs with paediatric indications should be put on label. She also iterated that to improve survival it is important to facilitate legislation regarding non-commercial clinical trials.

The presentation of Lydie Meheus can be found here.

The summary of the hearing is available here.

More information about the hearing on paediatric and rare cancers is here.