The Paediatric Regulation – a work in progress?

A guest blog by  Katharine Wright, Assistant Director, Nuffield Council on Bioethics

Katharine Wright

 

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.

 

 

 

A call for concrete action

Unite2Cure is delighted to be a partner in a multi-stakeholder call for concrete action to improve the availability of effective innovative cancer medicines for children and adolescents with cancer.  The call was launched at the event ‘Development of Paediatric Cancer Medicines – Speeding up Innovation, Saving Lives’. Although cancer remains the first cause of death by disease beyond one year of age; very few new medicines reach children and adolescents with cancer in Europe, even after the implementation of the 2007 EU Paediatric Medicine Regulation.

The proposals were presented at a yearly event by The European Society for Paediactric Oncology (SIOPE) www.siope.eu/activities  to mark International Childhood Cancer Day (15th February 2016).  The aim of the proposals is to suggest solutions to the current lack of drug development for children and teenagers with cancer.

©SIOPE & Joke Emmerechts photography

Chris Copland, one of Unite2Cure’s founding members, and Consumer Representative, National Cancer Research Institute (UK)

 

We are proud that Christopher Copland (UK), one of the founding members of Unite2Cure, spoke at the event of the urgent need to accelerate drug development for these young people.  He called for more attractive rewards for the pharmaceutical industry to develop innovative drugs for children’s malignancies; following the example of the “Creating Hope Act” system in the USA.

Also there was Unite2Cure partner Anne Goeres (Lu) from the Foundation Kriibskrank Kanner.  She stressed the need to join forces with the rare diseases’ community in advocating better treatments.

©SIOPE & Joke Emmerechts photography

Anne Goeres, one of Unite2Cure’s founding members, Fondatioun Kriibskrank Kanner (Lu)

 

 

Member of the European Parliament Glenis Willmott (S&D, UK), a strenuous supporter of the causes of the childhood cancer community over the past years, hosted and introduced the event. Martin Schrappe (DE), President of SIOPE, presented the ‘SIOPE-ITCC-CDDF Multistakeholder Platform’, created in 2013 by representatives of parent/patient advocates, academia, industry and regulators. The Platform works on proposals to increase innovative drug development that are regularly presented to decision-makers during a series of Paediatric Oncology Conferences, the last of which led to three stakeholders’ joint proposals to improve the effectiveness of the Regulation’s implementation.

Unite2Cure is a parent-led movement which aims to mobilise the parent/child/NGO voice to push urgently for better treatments and better access to treatments for young people with cancer.  The  current status quo means that:

– 6,000 children and teenagers die of cancer in Europe each year

– less than 1 in 10 of children with relapsed terminal cancer has access to  innovative new treatments.

– whilst progress has been made; many high risk cancers have not seen any therapeutic improvements in the past two decades

– childhood/teen cancers are largely neglected by industry

– current legislation allows waivers; even when science suggests a potential use

– EMA figures show that basically no drugs are developed for the high risk cancers.

If you care – join our discussion; sign our petition, or talk to us nicole@scobie.com   or deborahjanebinner@gmail.com

Time to accelerate

 

Last week several parents, survivors and patient advocates from Europe and the U.S. united in Brussels for the CDDF-ITCC-SIOPE 4th Paediatric Oncology Conference. The title of this year’s meeting was: Accelerating the Development of New Oncology Drugs for Children and Adolescents.

Debbie Binner of Create for Chloë presented the Unite2Cure movement along with Chris Copland, Consumer Representative, National Cancer Research Institute and Nicole Scobie of  Zoé4life. The presentation was very well received, and as Debbie explained later, the conference ended with a general sense of possibility. “Believe me the first CDDF conference was very very different to this one.  Us parents were fighting to be heard – and now there is such a sense of integration and collaboration and a real sense that we are making some progress.  Although obviously there is a long long way to go.”

 

A few key moments:

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Patricia Blanc presents

Patricia Blanc of Imagine for Margo‘s presentation on new incentives for pharmaceutical companies to invest in paediatric research was brilliant. Her voice is powerful and the work of her Working Group on this project has moved forward with an action plan. Nancy Goodman of KidsVCancer gave an excellent and comprehensive update on the successful US Creating Hope Act regulation.

Angela Polanco of Bethany’s Wish participated actively in a round table discussion on the new incentives topic, and explained that parents want to be involved from the very beginning in the process of approving paediatric investigation plans and participating in the development of clinical trials.

 

Chris Copland spoke up several times, passionately advocating for change to the Paediatric Medicines Regulation. He and Anne will be speaking on behalf of Unite2Cure today, January 27th in front of the European Parliament at an event organized by SIOPE in honour of International Childhood Cancer Awareness Day. This influential awareness raising meeting entitled ‘Speeding up Innovation, Saving Lives’ will be hosted by  MEP Ms Glenis Willmott. The goal is to address the development of paediatric cancer medicines and an enabling legislative and regulatory environment. In plain words: we are asking for very specific changes to the regulations NOW.

 

Leona Knox, of Solving Kids Cancer-Europe spoke with a powerful message, summarizing her feelings about the 2 day conference. “I am one of the parents here who won’t be going home to my son tonight. Oscar died, not because he had cancer, but because he ran out of treatment options. We parents have come here on our own time, and have worked so hard to make sure we understand what you have presented.” She told the audience of mostly pharmaceutical company representatives, pediatric oncologists and regulators. “Our goal is to save kids’ lives. We are working so hard to come up with solutions, and I just want to ask you all, to please help us fix this, whatever it takes.”

Both Samantha Schoolar of Bethany’s Wish and Jaap den Hartogh of VOKK spoke very clearly about the long term side effects survivors of childhood cancer experience and the need to speed up better treatments as well as research into late effects. “A real cure means being part of society once more,” explained Jaap, who called for new, better, and less toxic medicines as well as access to long-term follow up care for every survivor in Europe.

 

It was inspiring to hear Peter Adamson, the Chair of the Children’s Oncology Group stand up to remind the audience that the title of this conference was “Accelerating the Development of New Oncology Drugs for Children and Adolescents”, and that, having attended these conferences for the past several years, he did not want to just have the same conversations as every year. It was time to actually accelerate.

 

Gilles Vassal, past president of SIOPE and Director of Clinical and Translational Research at the Institut de Cancérologie Gustave-Roussy made a clear call to “change the mindset“.

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Change the mindset!

The summary of the decisions and plans as a result of the conference are, briefly:

-a strengthening of the  CDDF-ENCCA – ITCC-SIOPE platform with terms of reference, a website and a new name (to be announced!).

-position papers will be created by Working Group 1 on the issues of adolescent inclusion in adult phase one trials and on mechanism of action as a determinant for paediatric investigation plan requirements.

-a position paper is being drafted on Working Group 2’s proposals for new incentives

-a white paper will be put together by Working Group 3 regarding long term follow up for children receiving new drugs. This group met immediately after the conference to continue preparing this document.

Gilles Vassal emphasized that parents and survivors must continue to advocate for change for all of these plans to happen.

He ended the conference with one sentence, that summarizes the event, “We are even stronger if we work together.”

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The view over Brussels on the flight home

 

 

 

 

World’s Children Raise their Voices for Childhood Cancer in New Single

raiseyourvoice

Every day 700 children are diagnosed with cancer. It’s time to raise our voices.

Some of the most famous singing kids on YouTube are forming a supergroup to raise awareness about childhood cancer. And the best part? The children of the world are invited to join in!

At child4child.com kids can sing and record the chorus of the song, which will then be released on Feb. 15th, 2016—International Childhood Cancer Day. The song “We Are One” has been created by Christophe Beck, the composer for Disney’s smash hit Frozen.

Childhood Cancer International (CCI) is an umbrella organization currently representing 180 parent-led childhood cancer organizations in 90 countries. Today CCI has launched child4Child.com, the first global initiative where their member organizations join forces to raise awareness of childhood cancer. Of the 250,000 children that are diagnosed with cancer each year, about 90,000 of them will lose their lives to the disease, making cancer the number one non-communicable disease-related cause of death of children worldwide. The organization has now teamed up with some of the most talented singing kids on YouTube, many of them famous for their appearances on talent shows such as America’s Got Talent, The Voice Kids Australia and Britain’s Got Talent. Their performances have together reached over 300 million views on YouTube.

The new song “We Are One” is written by internationally acclaimed composer Christophe Beck, most known for creating the score to Disney’s smash hit Frozen.

“I’ve composed the scores to over a hundred films, including Hollywood blockbusters such as The Hangover, Ant-Man, and The Peanuts Movie. But this has by far been one of my most challenging assignments. Luckily I had my daughter Sophie by my side, a talented and precocious musician and songwriter herself, along with lyricist David Goldsmith, who wrote some beautiful words to inspire us. The three of us have been working on the song together and I am extraordinarily pleased with the results,” says Christophe Beck.

Children are invited to sing and record the chorus of the song on the child4child.com website starting January 11th. One month later on February 15th, the International Childhood Cancer Day, the song will be released on music streaming platforms such as Spotify and iTunes. The chorus of the song will contain all voices recorded on the website.

“With the song ”We Are One” we wish to honor all children of the world, particularly those in their courageous battle against cancer,” says Carmen Auste, the current Chair of CCI and the mother of a childhood cancer survivor.

Child Ambassadors for the campaign are Alexa Curtis (winner of The Voice Kids Australia 2014, Livvy Stubenrauch (the voice of Anna in Disney’s Frozen), Robbie Firmin (Britain’s Got Talent) and Aaralyn O’Neil (America’s Got Talent).

 

Children from around the world can visit the website from January 11th until early February. Then on February 15th, International Childhood Cancer Day, the chorus version of all of the children who had recorded themselves singing will be launched on global music platforms.

A music video will also be released on YouTube with clips of the children who have helped record the chorus.
Join the chorus of children around the world in solidarity with those battling cancer.

We are one.

We are strong.

And we will sing this song together

So sing along

And as one

We will rise

We won’t give up the fight until the night

Yields to the sun

We are one.

Two MEPs join the fight

On November 10th, 2015 Françoise Grossetête and Glenis Willmott, MEPs highly involved in the cause of children with cancer for many years, joined with Unite2Cure to CALL FOR IMMEDIATE ACTION to give more children and adolescents the prospect of a better life.

Their letter to Mr. Andriukaitis, Commissioner for Health and Food Safety, requests that the Commission immediately evaluate the situation and the application of the Paediatric Regulation, in order to be able to correct it as soon as possible.

As they state in their letter, today in Europe, children with cancer and other life-threatening diseases are being denied access to potentially life-saving treatments. This is a public health issue of prime importance. Cancer is the leading cause of death by disease in children across EuropeEach year, 35,000 children and adolescents are diagnosed with leukaemia or malignant solid tumours and 6000 of them die. Of the survivors, 40% will be left with severe long term side-effects which impact their daily life. For some conditions with a poor prognosis, only very limited improvements in treatments for children have been seen in recent decades.

The time for action is NOW.

To read the full letter to Commissioner Andriukaitis, click here

 

Taking care of business

Chris heads to the headquarters of the European Medicines Agency on behalf of Unite2Cure…

The brash banking centre of Canary Wharf, with its gleaming sky-high towers, is the unlikely location for the headquarters of that most decorous of institutions, the European Medicines Agency.  Standing between the murky waters of the Thames and Barclays HQ, the EMA gleams like a beacon for health rather than wealth, right in the heart of London’s bonus-driven business district.

The business I was to witness there, though, was of an altogether different kind.

Continue reading…

A response to our article

On November 23rd, Unite2Cure posted an article entitled We need to move the lines, which was our message to the European Commission. Written by Chris Copland on behalf of Unite2Cure, the letter was addressed to Ms Juelicher, who is Head of Unit, Medicinal Products – Authorisations, European Medicines Agency, as well as to Commissioner Andriukaitis, Commissioner for Health and Food Safety, European Commission.

In it we describe how we found the Commission’s position, that the concerns raised by Mrs Wilmott and Mme Grossetete during the  ‘Exchange of Views with the European Commission and EMA on the Policy on the Conditions for a Paediatric Investigation Plan/Waiver.’  on November 10, should be put aside until 2017, a sadly complacent one.

On December 17th we received a reply. Unfortunately, it does not address our concerns but is simply a copy of a letter sent to Mrs Wilmott and Mme Grossetete. We are disappointed in this response.

Response from Ms. Jülicher

Reply to Ms Grossetête and Ms Willmott,

We have replied to their letter as follows and hope to hear more soon.

 

Dear Ms Julicher

Thank you for your response to our message. We were, however, disappointed that you simply referred to your open letter to Ms Wilmott, MEP and did not provide us with a direct reply.  Although the letter was on associated themes, it did not address specific points we had made, for example about orphan illnesses. We, however, would like to respond here to the points you made, in particular your assessment of whether the Paediatric Regulation is functioning as effectively as it might.

As you point out, what makes the Paediatric Regulation different is that it does not just offer incentives, it places a requirement on companies to conduct studies into children. The effectiveness of this approach has been demonstrated in recent years but, unfortunately, it has not achieved its potential with regard to the most deadly of young people’s illnesses cancer.

You assert that the Regulation allows companies to propose paediatric plans for cancer on their own initiative, and it is true that on occasion this has happened. However, the track record, over decades, of companies voluntarily engaging with paediatric research is not an encouraging one (see Raymond and Herold). On the other hand, their willingness to avoid requirements by resorting to the loophole of the waiver system has been clearly demonstrated. As has been frequently related, during the first five years of the Regulation, over half of the adult drugs with a significant potential for children’s cancers had waivers requested and granted (see Vassal below).

You paint a positive picture by referring to the recent statistics supplied to you by the EMA. As we have not seen this data, we cannot respond to your interpretation in detail. However, we note that you place emphasis on the number of programmes that ‘target a condition where most patients are in the paediatric age range.’ You do not clarify whether, in these cases, a PIP has proceeded because there is a common condition for both adult and child or because companies have simply opted not to apply for a waiver. Either way, this ignores the simple fact that ‘’PDCO has to grant a waiver if the disease only occurs in adults.’ (EMA)

This is the central weakness of the legislation with regard to cancer, a disease which manifests itself differently in adults and children but still derives from a common biology. As Gilles Vassal has reported, 90% of the drugs successfully given to young people with cancer over the past 40 years have been used with different tumour types in adults. Ultimately, the legislation can only be made consistent and genuinely productive by basing the requirement to produce a Paediatric Investigation Plan on the biology that underlies adult cancers rather than simply the tumour type.

We acknowledge that some companies may conduct a PIP they are not required to and which may involve pursuing the mechanism of action. This may be the result of altruism or, more likely, because of the incentives the legislation offers. However, this is unlikely to generate research in anything more than a haphazard fashion. As parents, we expect industry to conduct paediatric research systematically and as a matter of course – our children deserve no less. This is why the Paediatric Regulation’s emphasis on requirements, which you yourself accept is what makes it different, is so important. The point is such obligations must be applied consistently and correspond with our current understanding of biological science.

It is of course heartening to read of the oncology PIPs currently proceeding, but, after ten years, these are still in relatively small numbers when one considers the scale of the problem to be overcome – 6,000 deaths of young people in Europe every year from cancer.

The urgency of the situation is distilled in the words of one parent:

‘It is inconceivable in this day and age that, globally, there is no drug that can cure my daughter’

This is the view of the nearly 1500 people who have signed our petition and of the many prominent professionals and associations (SIOPE included) that have come out in our support. If one reads the literature, there is a clear consensus in the professional community of the changes that are necessary to put the regulation on track and of the urgency of doing so. As members of the CDDF ITCC ENCCA SIOPE Paediatric Platform concluded at our conference this time last year: Waiting is not an option.

We look forward to a productive engagement with ENVI in the New Year. In the meantime, may we wish you and your staff a Happy Christmas.

Regards,

Christopher Copland

on behalf of Unite2Cure

 

 

 

‘We need to move the lines.’

Our message to the European Commission.

 

Ms Juelicher,

Commissioner Andriukaitis,

I am contacting you on behalf of Unite2Cure, an international network of families and supporters of children with cancer. A number of us watched with interest the video broadcast on 10 November of the ‘Exchange of Views with the European Commission and EMA on the Policy on the Conditions for a Paediatric Investigation Plan/Waiver.’  I would like to respond here to the statements Ms Juelicher made on behalf of the Commission at the conclusion of the meeting.

I would stress, at the outset, that we very much support the points Ms Wilmott’s representative made and were heartened that Mme Grosstete  called for action without delay. However, we found the Commission’s position, that the concerns raised should be put aside until 2017, a sadly complacent one. To clarify why, I respond below to each of the points Ms Juelicher made to justify this approach.  (Ms Juelicher’s words are transcribed in italics.)

Any regulatory framework has to provide a balance between promoting research (for children) and not delaying access to new medicines to adult.

 The report from the Commission to the European Parliament has, in fact, discounted this as an issue.

‘Studies prior to the adoption of the Regulation suggested a theoretical risk that the requirements for research in children could lengthen the overall drug development process. The Regulation has met this risk head-on… Experience… suggests that the risk of delays in the processing of adult applications is minimal.’[1]

One fact I would like to recall is that many of the paediatric cancers are rare diseases and so they do benefit from even more incentives for rare diseases.

Presumably, here ‘orphan’ drug legislation is being referred to. However, as the Institute of Cancer Research very recently pointed out:

‘Orphan drug designation has not proved effective at providing financial incentives for companies to develop drugs solely for paediatric cancers. No cancer drugs have gone through this process purely for childhood cancers, indicating that companies do not regard it as financially attractive.’[2]

I would like to reply to a suggestion to move very quickly onto a review of the Regulation before 2017. Development of medicinal products is unfortunately a lengthy process. Concluding at this moment in time on benefits and full effects of the Regulation is probably a bit premature. So we, as a Commission, move towards this 2017 review date in particular because … we need more comprehensive evidence before we conclude on the suitability or otherwise of the regulation.

 With regard to children’s’ cancers, it has been clear for quite some time that the Regulation is insufficiently effective. As early as 2012, the EMA itself drew attention to ‘therapeutic areas such as paediatric oncology where little progress has been made in the last five years in part due to the difference in clinical conditions between adults and children.’[3]

 The passage of time will not alter this situation because the problem exists at the outset of the process. The ‘loophole’ of class waivers means that, in case after case, Paediatric Investigation Plans (PIPs) with significant potential for children’s cancers simply never get started. As Ms Wilmott’s representative reminded us, of the twenty six adult drugs with a potential relevance to childhood cancers developed in the first five years of the Regulation, over half were granted waivers. The fact that the development of drugs is a lengthy process means that prevarication on establishing a workable process pushes the identification of viable therapies further and further beyond the horizon.

To conclude, we do not share that the PMR has not brought about tangible benefits. Just the opposite; there have been impressive developments in the meantime.

 The benefits the PMR can bring to the paediatric population in general have indeed been demonstrated. This is why it is all the more urgent that the routine waiver of PIPs for paediatric oncology becomes a thing of the past and children with cancer are given access to the great potential that the new generation of drugs offer .

During September, Childhood Cancer Awareness Month, Unite2Cure, set itself the task of raising awareness of the shortcomings in research into children’s cancer and of the specific issues around the Paediatric Regulation.  As a result, we are now in a position to present to you formally, as the representatives of the Commission, the results of our e-petition calling for immediate action. Please open the link below:

https://unite2cure.org/wall-of-support/

Signatories include many of Europe’s leading paediatric oncologists. Gilles Vassal, for example, Chair of the European Society of Paediatric Oncology, writes:

I sign the petition because we need to move the lines, to create a new mindset and a strong momentum that will speed innovation for children and adolescents.

Most supporters, though, are ordinary people who, like us, have had direct experience of childhood cancer. One parent of a boy with an ‘orphan’ cancer, for example, makes this statement:

We need better methods and regulations to support research and development of new therapies. Now.

This petition provides  well over a thousand reasons for you to reflect upon whether the European Commission is doing all it can to speed better therapies to children with this most intractable of  illnesses.

We were disappointed to see so few people in the chamber at the ENVI meeting on Tuesday, and we noted that the proceedings were brought to a close ahead of schedule. Unite2Cure, however, has demonstrated that in Europe and beyond, there is a genuine interest in this issue and a heartfelt desire for change. This is something we are determined to keep before the public.

We would therefore greatly appreciate a reply from the Commission to the petition and this message.  We assure you we will relay your response to all of our supporters.

Yours sincerely

Christopher Copland

on behalf of Unite2Cure

 

[1] European Commission (2013) Better Medicines for Children / From Concept to Reality: General Report on experience acquired as a result of the application of Regulation (EC) n° 1901/2006 on medicinal products for paediatric use.

 

[2] Institute of Cancer Research (2014) Early-stage clinical trials of cancer drugs for children. ICR: London

 

[3] EMA (2012) 5-year Report to the European Commission General on the experience acquired as a result of the application of the Paediatric Regulation.

A Disappointing Response: The Committee on the Environment, Public Health and Food Safety (ENVI)’s meeting with the European Commission and European Medicines Agency

The Committee on the Environment, Public Health and Food Safety (ENVI) met on November 10th at the European parliament for an exchange of views with the European Commission and European Medicines Agency (EMA) on the “Policy on the Conditions for a Paedriatic Investigation Plan/Waiver”. In the light of the disappointing response, it is clear that we need to continue to fight hard for important changes to save children’s lives.

The action from the initial meeting on 16th June was for the EMA to respond to a question about the implementation of Clause 11b in the Paediatric Regulation, a clause determining how a waiver is still issued based on disease as opposed to mechanism of action. Scientific understanding is such that mechanism of action is the way forward and the basis for development of new treatments for diseases.

Mme Grossetête, French MEP rapporteur for the Pediatric Regulation and Mrs Willmott, UK MEP rapporteur for the Clinical Trial Regulation presented their strong requests for an urgent review of the class waiver system to the Pediatric Medicines Regulation.

Despite Mme Grossetête’s repeated request for urgent action, the European Commission maintained its position that the Regulation need not be reviewed before the 2017 planned review.

Cancer is the leading cause of death by disease in children across Europe. Each year, 35,000 children and adolescents are diagnosed with cancer, and 6000 of them will die. Of the survivors, 40% will be left with severe long-term side-effects which impact their daily life.

Unite2Cure calls for immediate change to European Paediatric Medicine Regulation to save more lives of children and teens with cancer.

The time for action is NOW.

U2C in Vienna: the European Bone Sarcoma Meeting

As a parent rep. in the Euro-Ewing’s Consortium, I was invited to attend this networking meeting in September and decided to use this opportunity to bring Unite2Cure to the group’s attention.

Thanks to Stefan Bielack for organising a friendly, inclusive and very informative conference. Not convinced by the 8 a.m. start on the second day, though! 🙂

There was a good range of sessions, including some on research at very early stages, which gave a glimpse of what the future might hold. With regard to Ewing’s Sarcoma, what stuck in my mind was something Bass Hassan of the Oxford Cancer Centre said:

‘There are two agents with exquisite sensitivity: PARPi and IGF-1R’

These were targets that cropped up in session after session and it was heartening to learn from Sandra Strauss of UCL that a Ewing Sarcoma PARPi Consortium has been set up in collaboration with SARC, the American charity. As a parent representative on the Euro-Ewing’s 2012 trial, I find it disappointing that promising new agents like PARPi do not yet form a part of our flagship trial. Although, novel agents might be integrated into EE 2012 in the future, this will have to wait until one of the conventional chemotherapy arms is dropped.

The five minutes I had to present the Unite2Cure initiative prompted a positive response from delegates. U2C aims to amend European law so that there are strong obligations and incentives on drug companies to conduct research into children’s cancers. I was pleased to see that many delegates, like Bernadette Brennan and Martin McCabe, signed our e-petition over the following week.

As predicted, our petition easily smashed its target of 1000 signatures by the end of September, Childhood Cancer Awareness month. For me, what has been compelling, though, is not the number of signatories but the powerful stories almost all of them have had to tell, like this one from a Director in the USA.

 “My son has recurrent Ewings Sarcoma. Because it is an orphan cancer little progress is being made to fight it. We need better methods and regulations to support research and development of new therapies. Now. ”

Article submitted by Chris Copland, Consumer Representative, National Cancer Research Institute and Unite2Cure founding member.

Additional information

Ewing’s Sarcoma: a primary bone cancer that tends to strike adolescents between 10 and 20 years old.

Euro-Ewing’s Consortium:  International Clinical Trials to Improve Survival from Ewing Sarcoma (EEC project) is a University College London (UCL) coordinated collaborative project involving 20 European partners, which has recently been awarded funding through the European Union’s Framework Programme 7 (FP7).