National Institute for Health and Care Excellence (NICE) has recommended dinutuximab beta for treatment of high-risk neuroblastoma – a rare type of cancer that mainly affects children and young people – in final draft technology appraisal guidance published today.
It will be given to patients aged 12 months or over whose disease has at least partially responded to first stage chemotherapy, followed by myeloablative therapy and stem cell transplant but only if they have not received anti-GD2 immunotherapy.
The drug must also be provided by the company according to the commercial arrangement struck with the NHS, for treatment to be approved.
Evidence suggests dinutuximab beta (Qarziba, EUSA Pharma) increases overall survival compared with current treatments. While there is uncertainty around the long-term clinical benefit of the drug, the appraisal committee believed the potential survival gain offered by its use was substantial.
The committee took into account other factors, including health-related benefits not captured, the rarity and severity of the disease and the potential to increase survival for children with high-risk neuroblastoma. This allowed the NICE appraisal committee to recommend dinutuximab beta.
An agreement has been reached with EUSA Pharma to allow the drug to be made available to the NHS at a confidential discounted price. The average list cost of a course of treatment for a three year old is £152,200.
Meindert Boysen, director for the NICE Centre for Health Technology Evaluation, said: “We are pleased to be able to recommend dinutuximab beta. It is an important treatment option for children and young-people with high-risk neuroblastoma in particular, and has shown the potential to increase their survival.
“We are grateful to the company for responding to our evaluation by setting a price which is cost-effective to the NHS.
“We welcome this new and effective treatment option.”
Neuroblastoma is a cancer that develops from specialised nerve cells called „neuroblasts‟, which are left behind after a baby‟s development. It is most common in children under the age of five, and is estimated to affect 100 children each year in the UK.
Treatments for high-risk neuroblastoma include chemotherapy, radiotherapy, stem cell transplant, surgery and isotretinoin.
The main aim of current treatment is to extend survival, but experts have called for a cure. Up until 2009, maintenance therapy with isotretinoin was considered standard care in the NHS for people with high-risk neuroblastoma. Since then, dinutuximab beta has been available through a clinical trial and free supply from the company.
Joint statement in response to NICE’s decision to approve anti-GD2 antibody therapy dinutuximab beta for NHS clinical use.
From Solving Kids’ Cancer, Neuroblastoma UK, the Children’s Cancer and Leukaemia group, JACK, The Bradley Lowery Foundation, Hugs from Henry, Christopher’s Smile, Niamh’s Next Steps and Smile with Siddy, Grace Kelly Ladybird Trust, Ruperts Revenge Trust, Stacey’s Smiles, Harvey Hext Trust, George and the Giant Pledge and Abbie’s Army.
Charities that support children with high-risk neuroblastoma and their families, as well as other childhood cancers jointly welcomed today‟s decision to recommend a life-saving drug called dinutuximab beta for use on the NHS.
Representing all of the charities, Stephen Richards, Chief Executive of Solving Kids‟ Cancer, said: “We are absolutely delighted that the right decision has been made to benefit the lives of children with neuroblastoma. Parents, charities and clinicians have together helped bring about this positive result.
Families now know their very sick child will be able to have the best treatment that gives the greatest chance of long-term survival here in the UK.
We’ve been fighting within a complex system for the last three years, with the expertise of parents who have devastatingly lost their own children and doctors dedicated to improving research and care. Thank you to everyone who has helped give our sick and vulnerable children a voice.
We‟d like to acknowledge the work of those involved – including NHS England, NICE and EUSA Pharma – in coming together to agree this positive outcome. And we hope there is an opportunity to reflect on the way future treatments for seriously ill and vulnerable children are appraised.”
