Unite2cure was well-represented this October at the Better Medicines for Children conference, hosted by the European Forum for Good Clinical Practice and the Drug Information Association. Both Delphine Heenen and Chris Copland took part and Chris had the opportunity to present on the FAIR Trials age-inclusive research campaign. A colleague from the FAIR Trials working group, Christina Bucci-Rechtweg, of Novartis, also made headway with EFGCP colleagues in planning a position paper on age-inclusive research, with guidelines for ethics committees. The idea of FAIR Trials was well-received and Chris was invited by one of the delegates to speak at a workshop on paediatric oncology at Astra Zeneca later in the autumn
Unite2cure has been busy campaigning in the UK this week.
Our message has come across loud and clear: across the word we need better access to newer and kinder drugs for kids with cancer.
We must also immediately ditch lower age ranges for adult trials so that more kids can get a change of better treatments.
6,000 children die of cancer each year in Europe. It is the biggest killer by disease in the under 25 age range. We must unite for change.
The UK media has got our message loud and clear. Firstly there was an hour long documentary on teenage cancer from the BBC’s flagship scientific programme Horizon. This was followed by articles in the national UK press – all highlighting the need for better drugs for these children.
Debbie Binner is one of the founding members of Unite2Cure
Here are links to the press coverage and the BBC Horizon full programme:
An article in Cancerworld magazine by journalist Sophie Fessl, summarizes well the issues Unite2Cure is raising.
The chances of surviving a childhood cancer have changed very little over the past two decades. Sophie Fessl talked to parents, doctors, regulators and researchers about what has to be done to address this disastrous impasse. “It feels odd to say this, but Elliot is one of the lucky ones.” The comment comes from Nicole Scobie (photo, above, with Elliot), and refers to her son, who was only four years old when he was diagnosed with a stage IV Wilm’s tumour. His left kidney was engulfed by a huge cancerous mass and his lungs were full of metastases. The heart-stopping diagnosis was just the start of a rollercoaster of emotions, hospital stays and exhaustion. But this is a story with hope: Elliot responded well to chemotherapy. He went into remission after 10 months of treatment, and has remained so for the past four years.
“At least for his cancer, there is a treatment that works,” says Nicole. Not all the children she and her son befriended during their long stay at the Lausanne University Hospital’s children’s cancer ward can consider themselves as ‘lucky’. Elliot became close friends with Zoe, a little girl battling an aggressive neuroblastoma. But while Elliot’s prospects looked good, for Zoe, the odds were stacked against her. In the end, there was nothing her medical team could do: Zoe died in her mother’s arms aged four.
The difference between Elliot and Zoe? Elliot had a type of cancer that has been successfully treated for decades, being one of the first childhood cancers – alongside acute lymphoblastic leukaemia – to benefit from the chemotherapies pioneered by Sydney Farber back in the 1950s. His is part of the celebrated success story that saw cancer survival rates among children increase from 10% to 80% over 50 years.
Zoe, by contrast, had a type of childhood cancer that remains fatal in the majority of cases. Her story is shared by 6,000 children and young people under 24 who are still dying of cancer each year in Europe. For parents like Nicole Scobie, who’ve seen their child’s life in the balance, that is a heartbreaking statistic. But there’s a worse one – the mortality rate from childhood cancers has barely changed over the past 16 years.
Last year, Scobie was one of a large group of parents and advocacy organisations that got together to found Unite2Cure, an advocacy organisation that aims to kickstart progress again.
UNITE2CURE at the European Commission to meet with Health Commisioner Vytenis Andriukaitis: Changes are needed to the Pediatric Medecine Regulation
An excellent three-day event in Belgrade organized by Childhood Cancer International (CCI) Europe ended on a positive and motivated note for the several Unite2Cure members who attended. The general mood was that the message had been understood: the time for action is now!
The Unite2Cure movement was presented in a one hour interactive session by Chris Copland, Anne Goeres and Nicole Scobie.
“We wanted them to really hear what we were saying, really understand the message.” explains Nicole Scobie, from Zoé4life, Switzerland. “It was an early morning session after a late night out the evening before, and we knew people would be tired. We felt if we started out with a long power point on this difficult subject (paediatric medicines regulations), we would lose the audience. We decided to use humour and small working groups to get people talking and thinking about these issues.”
“Not everyone in the audience is a native English speaker,” adds Anne Goeres, from Fondatioun Kriibskrank Kanner, Luxembourg. “We realized that we needed to make sure we used simple, clear terms that everyone could understand.”
When Chris Copland, a teacher from the UK and Unite2Cure founding member began talking to the attentive audience about “loopholes” in the law, Anne quickly broke in to point out that she did not know this term. Chris was ready with his cable, to actually build a real “loophole” and show everyone how there is a hole in the law which allows pharmaceutical companies to avoid their obligations regarding child cancer.
Although the presentation was deliberately entertaining, the message was crystal clear: we must take action now if we are to have our voice heard. The review of the Paediatric Medicines Regulation is due in 2017 – this means we must act now.
Anne, Chris and Nicole ended their presentation with a call for action:
-become a Unite2Cure ambassador in your country
-mobilize your MEP and your minister of Health
-sign a “golden letter” to the commissioner of Health to the European Union.
The golden letters had been prepared ahead of time, with a space for each participant to add in his or her own message within Unite2Cure’s call for change to the PMR. Each letter was then sealed in a golden envelope, which were collected and will be presented to the Commissioner at Unite2Cure’s meeting with him in May.
If you would like to send a “golden letter”, please click here, download the letter, fill in your information, and email it to: email@example.com
If you would like more information on becoming a Unite2Cure ambassador in your country, or in supporting the movement, send us an email at firstname.lastname@example.org
A guest blog by Katharine Wright,Nuffield Council on Bioethics
Back in 2013, the UK-based Nuffield Council on Bioethics set up a working party to look at the ethical issues arising out of children’s involvement in clinical research. The working party’s subsequent report, drawing on the contributions of over 500 children and young people, parents and professionals, was published in May 2015, and its recommendations are made accessible in both a magazine format and as an animation.
The working party tackled not only the thorny issue of how children and young people can ethically be recruited to research, but also the more hidden ethical challenges that arise in connection with the ways in which research is prioritised and developed in the first place. We started from the premise that ‘good’ research (a shorthand for ethically robust and well-designed research that asks questions that are important for the health of children) should be seen as intrinsically good, and as a natural and necessary part of any healthcare system. Building on this point, we made the strong claim that it is not an ethically neutral act to say ‘no’ to research proposals that meet these requirements and might potentially lead to better health care for children. There is similarly a strong ethical imperative actively to promote such research – and as Unite2Cure highlights, the European Paediatric Regulation which mandates the inclusion of children and young people in clinical trials, unless a waiver has been granted, has a critical role to play in this arena.
The working party felt that the European Medicines Agency and the Paediatric Committee (the European institutions responsible for implementing the Regulation) should be commended for the very positive and proactive approach they have taken to their regulatory role. They have used it not only simply to police the system established by the Regulation, but have also sought actively to promote effective, collaborative, research with children and young people through a variety of practical means.
Nevertheless, much more can and should still be done – and our report picked out in particular the class waiver system, whereby medicines targeting ‘adult-only’ conditions have been exempt from the requirement to include children and young people in trials. We argued that this system was clearly not working as originally intended and strongly urged the Paediatric Committee (PDCO) to complete its promised review of the class waiver system as quickly as possible. In particular, we argued that PDCO should ensure that where the ‘mechanism of action’ of a medicine is potentially relevant to children (for example for a different illness from that targeted in adults), research with children should go ahead – a recommendation echoed in Unite2Cure’s proposals for improving research and care for children with cancer.
We therefore regarded PDCO’s announcement in July 2015 that they had revised the class waiver list, revoking eight such waivers, and updating fifteen more, as a “welcome step towards further promoting research with children”. This means that there are now fewer conditions where companies developing new medicines are automatically exempted from any requirement to develop a ‘paediatric implementation plan’ (PIP) setting out how they will involve children and young people in the study. However, under the Regulation, they are still potentially entitled to apply to PDCO on a case-by-case basis, and apply for a product-specific waiver, on the grounds that the condition being targeted by the trial medication does not arise in children. Thus, while PDCO will now be directly involved in discussing such cases with companies, they still cannot actually require them to produce a PIP – unless and until the Regulation is changed to give PDCO these powers.
The ‘ten-year review’ of the 2006 Regulation is coming up soon, offering a perfect opportunity to review the thinking behind the waiver system, and to ensure that PDCO has the powers it needs. We have written to the European Commission to urge the ethical imperative of such a change. In the meantime we urge all sponsors of research to consider the potential benefits of their research to children’s health – and where appropriate to include children and young people in trials on a voluntary basis.