Berlaymont EU commission building in Brussels. Credit: Flickr user Dimitris Avramopoulos (CC BY-SA 2.0)  

This letter was written to Jean-Claude Juncker, President of the European Commission by The Institute of Cancer Research, London, and its partner signatories, setting out our concerns on delays in reforming the EU Paediatric Regulation.

We are committed to supporting the European Commission’s efforts to improve access to medicines for children – but we are concerned that a golden opportunity has been missed to bring about change.

We believe that the failure to take immediate action to reform the EU Paediatric Regulation has worrying implications for the lives of young cancer patients. We offer any support we can provide in delivering changes to the regulation as soon as possible.

Only the European Commission has the power to reform the legislation governing development of new medicines. We call on you to use your considerable influence to deliver the changes that are needed as a matter of urgency.

We acknowledge that the Paediatric Regulation has delivered progress in the number of drugs overall that have entered paediatric early-stage clinical trials. But the picture is quite different, and much less positive, in the important case of children with cancer.

The Commission itself admits in its recent 10-year review of the regulation that children’s cancer therapies have not advanced at the same pace as adults. Indeed, Vytenis Andriukaitis, Commissioner for Health and Food Safety, has expressed deep upset at the lack of progress for children with cancer, and conceded that the regulation must ensure we can make more breakthroughs in paediatric oncology.

So it is extremely disappointing that the European Commission, rather than taking action now to correct the clear flaws in the current regulation, has instead proposed a two-year review into how it works with the rules governing orphan drugs.

As scientific institutions, charities and parents’ organisations, we do not believe that we can wait two years before acting. We are calling for urgent reform, to update the regulation and ensure children and young people have the same access to new cancer drugs as adults.

The current Paediatric Regulation could do much more to ensure that children benefit from the dramatic advances in treatment we are seeing for adult cancers.

Pharmaceutical companies are being given waivers from testing cancer drugs in children because they are targeting an adult cancer that does not occur in children – even though the drug may work in a way that could be effective against paediatric cancers.

The system of paediatric investigation plans setting out how drugs will be studied in children needs to be urgently revised to ensure cancer drugs developed for adults are tested in children whenever their mechanism of action suggests they could be effective.

Last year the European Parliament voted for a resolution urging the European Commission to consider making legislative revision of the Paediatric Regulation, in order to speed up the development of innovative new medicines for children. As you know, initiatives of this kind have already moved ahead in the US, where children now have more options for drug access and more choices for treatment than they do in Europe.

The resolution proposed that the regulation should be updated to ensure that a drug’s mechanism of action must be taken into account before any waiver is granted. We believe this simple change would have a dramatic impact on the number of clinical trials for children with cancer.

We were incredibly disappointed to see that the European Commission did not use the 10-year review as an opportunity to follow this recommendation from the European Parliament.

We urge you to use your influence to ensure the Commission revisits this decision, in order to grant children access to the innovative cancer drugs they so badly need. If there is a way we can work with you to deliver this much-needed change to the legislation then please let us know as we are keen to support any efforts to bring forward these changes sooner.

We cannot accept that outdated legislation should be allowed to deny thousands of children with cancer across Europe the treatments they so urgently need.

Signatories

Professor Paul Workman FRS FMedSci, Chief executive, The Institute of
Cancer Research, London

Professor Bobbie Farsides, Chair, Nuffield Council on Bioethics working
group on Children in clinical research: ethical issues

Jo and Andrew Williams, Founders, Lucas’s Legacy

Sian and Martin Waite, Founders, Elin Rose Appeal

Simon Fuller, Director of Services, Teenage Cancer Trust

Sara and David Wakeling, Founders, Alice’s Arc

Chris Copland, Founder member, Unite2Cure

Lola Manterola, Founder and President, CRIS Cancer Foundation

Nikki and Richard Bowdidge, Founders, The Tom Bowdidge Foundation

Mike Shaw, Trustee, Abbie’s Fund

Dr Jennifer Kelly, General practitioner and founder, Grace Kelly Ladybird Trust

Lynn and Lynn Lucas, Founders, The Chris Lucas Trust

Karen and Kevin Capel, Founders, Christopher’s Smile

Nigel Short, Trustee, Ewing’s Sarcoma Research Trust

Caroline Weston, Research and Policy Manager, CLIC Sargent

Professor Winette Van der Graaf, Professor of Personalised Onclogy, The Institute of Cancer Research, London

Professor Stan Kaye, Emeritus Professor, The Institute of Cancer Research, London