The National Institute for Health and Care Excellence (NICE) has today announced that it has upheld the appeal by Solving Kids’ Cancer against the Institute’s decision not to recommend dinutuximab for treating high-risk neuroblastoma. The decision on whether to recommend dinutuximab will now be referred back to the Appraisal Committee for reconsideration.
Solving Kids’ Cancer had argued that NICE had exceeded its powers and was in breach of the UN Convention on the Rights of the Child, and that using a 10-year cure point as the basis for the drug’s effectiveness was unreasonable given the evidence before it. In upholding the appeal on both grounds, the Panel concluded that:
“The Appraisal Committee should have the opportunity to consider whether there is anything particular to this patient group as children that should be taken into account in the appraisal, and if there is, to take it into account and to see whether the outcome of the appraisal is different, and if there is not, to say so with reasons.”
“The Committee should also review a range of cure points and associated ICERs between 5 and 10 years and should outline in the FAD the balanced reasoning for their choice.”
When the appeal against NICE’s decision not to fund this potentially life-saving drug in England and Wales was lodged in July 2016, it was the first time a charity had acted alone in challenging a Final Appraisal Determination (FAD). The charity subsequently presented its case at an oral hearing in September.
Responding to the Appeal Panel’s verdict Stephen Richards, Chief Executive of Solving Kids’ Cancer said:
“This is a crucial first-step in finally getting this life-saving treatment approved for use on the NHS. We remain absolutely committed, on behalf of UK children diagnosed with this devastating disease, to doing everything in our power to ensure this drug is made available. The alternative – that UK children are condemned to a reduced chance of survival – is simply unthinkable. By their own assessment, NICE agree that dinutuximab is a novel approach that saves the lives of some children and extends the lives of others. The Institute must now properly consider the unique contexts, challenges and considerations that exist in treating this rare and extremely vulnerable patient population.”
“This is the first time a charity has made a unilateral appeal against a NICE decision, and I am extremely proud to lead an organisation that is prepared to break the mould to fight for what it believes is right. We would also like to extend our sincerest thanks to Covington and Burling for taking on this case pro-bono, and without whose assistance this appeal could not have been taken forward in the manner it has. These children have no voice of their own, and we owe it to them to advocate, in the strongest possible terms, for a decision based on more than the reduction of their lives to a simple matter of pounds and pence.”
John Rogers, a scientist and the father of Stella who survived neuroblastoma after being treated with dinutuximab in the US, was one of the charity’s representatives at the Appeal Hearing. He said:
“Being a small charity with limited resources, we certainly felt like underdogs going into the Appeal Hearing. However, we have right on our side. Our children deserve to have the best chance of long-term survival, and this is what dinutuximab gives them. The Appeal Panel was fair, listened carefully to the arguments we put before them, and concluded that the Appraisal Committee must take into account that this disease affects mainly young children, and that it must more carefully consider how best to interpret the available scientific evidence.”
“It’s ludicrous to think that a drug developed to treat 30 or 40 children each year suffering from a deadly paediatric cancer should be assessed in the same way as drugs for diabetes or heart disease. What we have come to learn through this whole appeal process is that a more appropriate mechanism for assessing this type of treatment, in this type of patient population, is NICE’s Highly-Specialised Technology (HST) process. This is what should have been used from the outset. Unfortunately, it was not within the Appeal Panel’s remit to make such a determination, and so the fight goes on.”
The NICE ruling comes as an online petition signed by 116,000 members of the public, calling on the government to make more money available in the fight against child cancer was debated in Parliament on Monday.
NOTES TO EDITORS
- Solving Kids’ Cancer (Europe) is a charity bringing together children affected by cancer, their parents, families, researchers and health care professionals, to provide support, fund research and drive forward a cure for this devastating condition. For more information, please visit the website at solvingkidscancer.org.uk or Facebook page at www.facebook.com/solvingkidscancer.
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