‘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.