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.
on behalf of Unite2Cure