April 2022 decisions news release
Advice on three new medicines for use in conditions including a rare and fatal genetic condition affecting young children and a form of leukaemia has today (Monday 11 April) been published by the Scottish Medicines Consortium.
An initial assessment report has been published for atidarsagene autotemcel (Libmeldy) which can be used to treat children with a rare genetic condition called metachromatic leukodystrophy (MLD). This medicine has been assessed through the ultra-orphan pathway for medicines that treat extremely rare conditions. MLD causes damage to the nervous system and children with the condition rapidly become unable to walk, talk, swallow, see and hear. The condition is fatal and children usually only live for five to eight years. MLD impacts on the patient but also has a devastating impact on their families and carers. Atidarsagene autotemcel is a highly specialised stem cell treatment that is given as a single dose and could potentially allow children to develop physically to fully participate in everyday life and attend school. The available clinical evidence for atidarsagene autotemcel is, however, limited and despite a confidential discount provided by the company, the cost in relation to the health benefits remains high. The company is now required to provide a plan detailing how further data on the effects of the medicine, including those on the patient and carer lived experience, will be collected. Atidarsagene autotemcel will then be available for three years while the information is gathered. Following this, SMC will review the evidence and make a decision on routine availability in NHSScotland.
Further information on the ultra-orphan pathway can be found in Scottish Government guidance.
Venetoclax (Venclyxto) was accepted for the treatment of acute myeloid leukaemia (AML), a cancer of the white blood cells. Used in conjunction with a chemotherapy medicine, venetoclax can be used in patients newly diagnosed with AML who are unable to have intensive chemotherapy. Venetoclax was considered through SMC’s Patient and Clinician Engagement (PACE) process, which is used for medicines for end of life and rare conditions. In the PACE meeting, participants explained the symptoms of the condition which can include fatigue, feeling weak, bleeding and bruising as well as pain and difficulty moving. The addition of venetoclax to current treatment may increase overall survival and reduce symptoms therefore improving a patient’s wellbeing and quality of life.
Upadacitinib (Rinvoq) was accepted for the treatment of adults and adolescents with moderate-to-severe atopic dermatitis, a chronic inflammatory skin condition. The itch can be intense, relentless and unbearable and constant scratching causes the skin to split and bleed, leaving it open to infection. Upadacitinib is an additional treatment option that can improve symptoms and allow patients a better quality of life. This treatment is taken orally and may help patients better manage their care.
SMC Chairman Mark MacGregor said:
“Metachromatic leukodystrophy is a devastating hereditary disorder. The patient group representatives shared powerful personal testimony with the committee, describing the extent of disability the condition causes and its life changing impact on patients and families. Atidarsagene autotemcel will be available in NHSScotland through the ultra-orphan pathway. This will allow patients with MLD to access treatment while more information on its clinical effectiveness is gained. Scottish Government will announce when this medicine will be available for prescribing. The cost is, however, extremely high. It is essential that the pharmaceutical industry works to improve its efficiency in developing these life changing therapies if the costs are to be sustainable for health systems around the world.”
“From the evidence given by patients and clinicians in the PACE meeting, we know that AML can cause a range of distressing symptoms. Adding venetoclax to current treatment may have benefits for some patients and we know this decision will be welcomed.”
“Our decision on upadacitinib provides another treatment option for atopic dermatitis which may improve symptoms and provide patients with a better quality of life.”