January 2024 decisions news release
The Scottish Medicines Consortium (SMC), which advises on newly-licensed medicines for use by NHSScotland, has today (Monday, January 15) published advice on three medicines.
Burosumab (Crysvita®) was accepted for treating children (aged one to seventeen years), with X-linked hypophosphataemia (XLH). XLH is an extremely rare hereditary condition characterised by low levels of phosphate in the blood, which results in painful skeletal deformities.
In January 2020 burosumab was accepted for use in NHSScotland for three years, through the Scottish Government ultra-orphan pathway, while further evidence, including about the effects of the medicine on the lived experience of patients and carers was gathered. Following reassessment after this three-year period, it has now been accepted by SMC for routine use.
Pembrolizumab (Keytruda®) was accepted for treating adults with advanced cancers including endometrial cancer, colorectal cancer and gastric, small intestine or biliary cancer that have a rare mutation.
Belantamab mafodotin (Blenrep®) was not recommended for treating adults with advanced myeloma who have had at least four previous treatments.
SMC Chair Dr Scott Muir said: “We are very pleased to be able to accept these two new medicines for use by NHSScotland.
“Burosumab for treating children with XLH is the first of the medicines for extremely rare conditions to be accepted for routine use by SMC after assessment of additional evidence collected during the three years it was made available through the ultra-orphan pathway. Through the SMC Patient and Clinician Engagement process, the committee heard the difference this medicine has made to patients and their families.
“Pembrolizumab will provide a targeted treatment option for certain patients with advanced cancers.
“The committee was not able to accept belantamab mafodotin (Blenrep®) for treating adults with myeloma as the evidence provided by the company was not strong enough to satisfy the committee. We would welcome a resubmission when the company has had an opportunity to address the key clinical and cost effectiveness uncertainties highlighted.”