A medicine used to treat a rare cancer which occurs predominantly in very young children has been accepted by the Scottish Medicines Consortium (SMC) for routine use in NHS Scotland.

Dinutuximab beta (Qarziba) is used to treat neuroblastoma, a cancer of the nerve cells that is predominantly diagnosed in young children and has a high rate of mortality. Dinutuximab was accepted following consideration through the SMC’s Patient and Clinician Engagement (PACE) process, which is used for medicines to treat end of life and rare conditions.

In the PACE meeting, patient groups and clinicians spoke of tumours occurring in the neck, chest, abdomen or pelvis. Children can experience a range of distressing symptoms such as nausea and vomiting, bone pain, weight loss, bleeding, kidney damage, breathing problems and spinal compression leading to paralysis.

Dinutuximab can improve survival and may offer the potential for a more normal childhood. For those children who have not responded to initial treatment or who have relapsed after responding to initial treatment, it may delay the time until disease progression.

Negative appraisal for bladder cancer treatment

However, the committee was unable to accept atezolizumab (Tecentriq) for bladder cancer in patients who have already undergone chemotherapy. Atezolizumab was also not recommended after consideration through the PACE process. Despite the additional flexibility this process allows, the committee was unable to recommend it as there was too much uncertainty in the company’s evidence about both the clinical benefits of the medicine and its cost effectiveness.

Also not recommended was fampridine (Fampyra) for the improvement of walking in adults with multiple sclerosis (MS) and walking disability. The committee was unable to recommend fampridine as there was too much uncertainty around its value for money.

SMC Chairman Dr Alan MacDonald said: “I am pleased the committee has been able to accept dinutuximab for the treatment of neuroblastoma. Patients and clinicians who took part in our PACE meeting told us how devastating this condition can be for young children and their families, so we hope this will be a welcome decision.

“We were unable to accept atezolizumab as the company’s evidence about the clinical and cost effectiveness of the medicine was not strong enough.

“For fampridine, the committee did not recommend the medicine as there was too much uncertainty in the company’s evidence about its cost effectiveness.”