New drug to help Scottish children with rare form of cancer

A drug that could improve survival rates for children with a rare and “notoriously difficult to treat” form of cancer has been approved for use by the NHS in Scotland.

The Scottish Medicines Consortium has agreed Dinutuximab beta – also known as Qarziba – can be used to try to help youngsters with neuroblastoma.

Drug manufacturers said the decision could benefit about seven children a year.

The disease is a cancer of the nerve cells, which occurs mainly in very young children, with campaigners saying the SMC’s decision meant patients in Scotland would receive the same type of care as in other parts of the UK.

Neuroblastoma UK chairman Tony Heddon said: “This is fantastic news for the families in Scotland of newly diagnosed children with this cancer and ensures that they will receive the same standard of care as other children in the rest of the UK.”

He added: “High-risk neuroblastoma is a notoriously difficult to treat and aggressive cancer that leaves young patients and their families facing an uncertain future.

“Children battling this disease need all the help they can get and that includes access to treatments that may help improve their condition.

“We welcome this decision and would like to recognise the efforts made on all sides to ensure a successful outcome.

“The fact that more children will now be able to potentially benefit from this immunotherapy will provide much-needed reassurance for families affected across Scotland.”

Children with neuroblastoma can suffer a range of symptoms including nausea and vomiting, bone pain, weight loss, bleeding, kidney damage, breathing problems and spinal compression leading to paralysis.

But the drug can improve survival rates and, for those patients who have not responded to initial treatments, it may be able to delay the progression of the disease.

Lee Morley, CEO of the drug firm  EUSA Pharma, said: “We are delighted to have been able to work with the SMC and the neuroblastoma community to make Dinutuximab beta available to all eligible children who may benefit across Scotland.

“High-risk neuroblastoma is a devastating disease. While today’s approval highlights that progress is being made, much more still needs to be done to help those children and families affected.

“This is a long-term goal for EUSA Pharma and we remain committed to continuing our work to improve care in this disease across the UK and beyond.”

SMC chairman Dr Alan MacDonald said: “I am pleased the committee has been able to accept Dinutuximab for the treatment of neuroblastoma.”

He added doctors and patients had “told us how devastating this condition can be for young children and their families, so we hope this will be a welcome decision”.

The SMC rejected the routine use of a drug for bladder cancer patients, as well as one for MS sufferers.

In the case of atezolizumab, which can be used to treat bladder cancer patients who have already undergone chemotherapy, Dr Macdonald said the “company’s evidence about the clinical and cost effectiveness of the medicine was not strong enough”.

For fampridine, which can improve walking in adults with MS, he said: “The committee did not recommend the medicine as there was too much uncertainty in the company’s evidence about its cost effectiveness.”

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