November 2020 decisions news release

Advice on eight new medicines for use in conditions including breast cancer, lung cancer and osteoporosis has today (Monday 9 October) been published by the Scottish Medicines Consortium.

An initial assessment report has been published for volanesorsen (Waylivra), which can be used to treat familial chylomicronaemia syndrome (FCS). This medicine has been assessed through the ultra-orphan pathway for medicines which treat very rare conditions. FCS is a genetic condition that gives rise to high levels of fats called triglycerides in the blood. Excess fat builds up in various parts of the body and leads to symptoms including abdominal pain and acute pancreatitis (inflammation of the pancreas). The only current treatment option for this condition is a very highly restrictive diet and this is of limited benefit. Volanesorsen may reduce abdominal pain and episodes of severe pancreatitis and may improve the quality of personal and social life. The submitting pharmaceutical 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. Volanesorsen will then be available through NHSScotland 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 approach can be found in Scottish Government guidance.

Atezolizumab (Tecentriq) was accepted for the treatment of metastatic triple negative breast cancer following consideration through SMC’s Patient and Clinician Engagement (PACE) process, which is used for medicines to treat end of life and rare conditions. Through the PACE meeting, patient groups and clinicians spoke of the devastating impact that a diagnosis can bring as it is associated with poorer outcomes than other types of breast cancer, tends to affect younger women and there are currently limited treatment options. Atezolizumab, in combination with chemotherapy, may improve progression free survival and potentially overall survival, allowing patients valuable extra time with family and friends.

Atezolizumab was also accepted for the treatment of extensive stage small cell lung cancer (ES-SCLC), an aggressive form of cancer with poor prognosis. Participants in the PACE process highlighted how a diagnosis of ES-SCLC often happens at a late stage, when patients may only have weeks or months to live. ES-SCLC is severely debilitating, with symptoms including breathlessness, chest pain, fatigue and weight loss. Atezolizumab, in combination with chemotherapy, is well-tolerated and may offer patients with ES-SCLC the chance of improved progression free survival and overall survival.

The committee also accepted trabectedin (Yondelis) following consideration through the PACE process. Trabectedin can be used to treat advanced soft-tissue sarcoma, a type of cancer that develops from the soft, supporting tissues of the body. In the PACE meeting, patient groups and clinicians emphasised how sarcomas are a rare and diverse group of cancers which are difficult to diagnose. Patients may report a wide range of symptoms depending on the location of the primary tumour. Symptoms such as fatigue and pain may be moderate until there is a rapid decline in the later stages of disease. Current treatment options for advanced disease are limited. Trabectedin offers another treatment option which gives patients the opportunity of an extended period of stable disease.

Romosozumab (Evenity) was accepted for the treatment of severe osteoporosis in postmenopausal women. Osteoporosis is a disease that makes bones fragile. The medicine was accepted for the treatment of patients who have had a fracture due to osteoporosis and are at risk of a further fracture within two years. Romosozumab can reduce the risk of fractures and provides another treatment option for women with severe osteoporosis.

The committee was unable to accept patiromer (Veltassa) for the treatment of hyperkalaemia, a condition in which potassium levels in the blood are raised, leading to increased risk of cardiac arrhythmias. The committee was not satisfied with the company’s evidence on the cost effectiveness of the treatment.

SMC Vice Chairman Scott Muir said:

“The committee is pleased to be able to accept these medicines for use by NHSScotland.”

“Volanesorsen will now move to the next stage of the ultra-orphan pathway. This will allow patients with FCS to access treatment while more information on its clinical effectiveness is gained. Scottish Government will announce when this medicine will be available for prescribing in NHSScotland.”

“From the evidence given by patients clinicians in the PACE meeting, we know that our decision on atezolizumab for the treatment of triple negative breast cancer will be welcomed by patients and their families.”

“For those with ES-SCLC, our decision on atezolizumab provides them with the opportunity of some valuable extra time with family and friends.”

“Our decision on trabectedin for advanced soft tissue sarcoma provides another treatment option for patients which may allow their condition to be stabilised.”

“We were also pleased to accept romosozumab for osteoporosis, enabling patients to benefit from another treatment where others have not been suitable.”

“We were unable to accept patiromer for hyperkalaemia as the company’s evidence about the medicine’s benefits was not strong enough.”

In addition to these medicines, SMC has also accepted the following medicines through an expedited approach to minimise delay in patient access following the early phase of the COVID-19 pandemic. 

• darolutamide (Nubeqa) for the treatment of prostate cancer
• trastuzumab emtansine (Kadcyla) for the treatment of breast cancer.


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