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Joining Against Cancer in Kids

Cancer drug for children recommended after becoming cost effective for NHS patients

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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.”

Jack Brown

Jack Brown
Emergency Grant for Neuroblastoma FamiliesFunds have been made available by J-A-C-K and Solving Kids’ Cancer to help families in the UK affected by Neuroblastoma

An Emergency grant is available for Neuroblastoma families facing financial loss or increased costs because of Coronavirus measures.

Funds have been made available by J-A-C-K and Solving Kids’ Cancer to help families in the UK affected by Neuroblastoma (low, intermediate or high-risk), who face financial loss or increased costs due to the Coronavirus pandemic.

Guidelines have been issued by the Children’s Cancer and Leukaemia Group asking that families of children and young people on active cancer treatment, or within 6 months following the completion of treatment, stay at home for the next 12 weeks. These guidelines are being reviewed and updated on an ongoing basis. This is in line with the government guidance.

Parents / Guardians who are self-isolating alongside the child and that suffer financial loss or increased costs as a result may apply to the emergency fund for an initial grant towards their immediate subsistence costs.

Emergency grants of £250 are available now for those who are facing financial loss or increased costs as a result of the need of their child to stay home for the stated 12 weeks.

Additional support may be made available at a later stage for families facing acute financial loss because of the rapidly evolving public health crisis.

Criteria for accessing the grant is as follows;

  • You are the guardian of a child with Neuroblastoma (low, intermediate or high-risk) who is currently in active treatment, or within 6 months following the completion of treatment (at the point of application).
  • You have suffered financial loss or increased costs as a result of the need to self-isolate with your child which cannot be remedied by the emergency measures in place which can be found on the government website which is being continually updated. Also here.

To apply for the £250 emergency grant simply complete the form below and submit it by email to familygrant@solvingkidscancer.org.uk along with a copy of a letter or other documentary evidence from your Clinician or Medical institution which provides evidence of the active treatment of your child or treatment within the last six months (of the date of application).

We, at JACK, are thankful for the ability to provide money alongside SKC to the emergency fund for families. We are grateful to Solving Kids Cancer UK for administering the process and applications for the grant must be made via the email to Solving Kids Cancer.