Marta and I have been extremely moved by the extraordinarily generous and spontaneous offers of support which we have received since I posted the situation about Isita’s immunotherapy drug. We are currently working through a series of options. But please be reassured, if we need to call for any other kind of help we will certainly do so. Right now, for the sake not just of Isita but for all the other children involved we need to make sure that as many people as possible know what is going on, so that the requests we have for the government and the NHS are heard, understood and acted upon. So our request is that you forward this statement, or a link to this blog, to anyone whom you think could help, and that you post it, tweet and retweet it on social media.

Three requests for the government and the NHS

22 April 2018

In early January 2016, our daughter Isita, now aged six, was diagnosed with stage-four neuroblastoma, a rare and difficult to treat cancer which develops out of neural crest cells and affects about 50 children in this country each year. For the past 16 months she has gone through a difficult and sometimes extremely painful complex of treatment. She has spent much of this time at Great Ormond Street Hospital in London, where the care she has received has been exceptionally good.

Until late last year, all children in the UK diagnosed with high-risk neuroblastoma were given as part of their care a drug called Dinutuximab Beta, an artificial antibody which destroys neuroblastoma cells as if they were viruses. This immunotherapy treatment is vital in reducing the chances of relapse – the event feared most by every parent. The drug was given as part of a clinical trial, which Isita was included in until complications meant she no longer qualified.

In early February, Isita became the first of a new cohort of children to be given this drug outside the trial. The individual funding requests submitted to the NHS by the oncologists at Great Ormond Street for every child were refused and so EUSA Pharma, the company which manufactures the drug, agreed – on compassionate grounds – to supply it for free at its own cost and risk. Isita has now had two out of the five cycles which are necessary to complete the treatment.

On 11 April, EUSA Pharma delivered a dossier of evidence to the National Institute for Clinical Excellence (NICE) which will be used to decide whether the NHS will fund the treatment as part of its standard of care for treating high-risk neuroblastoma. Last Thursday – a few days after NICE accepted the dossier – my wife and I and the parents of four other children also receiving this drug at GOSH were called to a meeting with our children’s doctors who relayed the news that the company can no longer supply this drug for free. We do not blame it for this decision. The hospital has enough supplies to finish the current 10-day cycles being administered to the other children in Isita’s group. From then on, the drug will only become available on the NHS once more if NICE decides that it should be funded. It may take several weeks to issue its decision. Children around the country will soon be affected as other hospitals also run out.

This raises a prospect which the doctors say they have never previously encountered in paediatric medicine: the cancellation of vital treatment in the middle of its administration, not because it is ineffective or too dangerous, but because there is no money for it. We do not believe that this is in any way intended, nor that it is a precedent that the NHS or the government would wish to let stand.

We understand that NICE has an unenviable task, and that none of the funding requests that it has to adjudicate between are trivial. So, our first request is that it decides as quickly as possible to relieve our present uncertainty. Clearly, we would prefer the certainty that the drug will continue to be provided, to the certainty that it won’t. But if it is to be the latter, the sooner we know, the sooner we can try to organise alternative funding. Ideally there should be a three-week gap between the end of one cycle and the beginning of the next. Some delay is possible, but time is not on our side. This is not just important for the children currently receiving the drug, but for those who would normally have expected to start in the next few weeks, and for any child now diagnosed or about to be diagnosed with neuroblastoma. In the worst case, a lot of fund-raising will be necessary.

Our second request, to NICE, but also to the Secretary of State for Health Jeremy Hunt is to pay particular attention to the cruel position in which those children now getting the drug have been left. If NICE’s decision is positive no harm will come, but if negative, the chances of finding replacement funding for all the children in mere weeks are not good. The arguments for compassionate funding to prevent these children from being abandoned are surely very good.

Our third and final request is that the Secretary of State and the Government take note of the fact that the immunotherapy our children have been getting until now is part of the standard of care for treating high-risk neuroblastoma in the United States. We understand that everywhere else in Europe, health ministries routinely approve individual funding requests for this drug. Now that it has all the facts, we know that NICE must decide this question on the rational basis that underpins all its decisions, and that it must make the best of the resources at its command. But it is nevertheless the case that a negative decision would place the NHS’s standard of care for neuroblastoma at a lower level than in most nations we would like to compare ourselves to.