December 2020 decisions news release

Medicines which can be used to treat a form of blood cancer and a rare muscle disorder have today (Monday 7 December) been accepted by the Scottish Medicines Consortium (SMC) for use by NHSScotland.

Venetoclax (Venclyxto) was accepted for the treatment of certain patients with previously untreated chronic lymphocytic leukaemia (CLL). The medicine 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 highlighted that CLL is an incurable, relapsing blood cancer that tends to affect older adults. Patients begin to receive treatment when disease- related symptoms are present, such as large and painful lymph nodes or reductions in their red blood cell or platelet counts, which can lead to substantial fatigue and difficulties carrying out activities of daily living. They may not be able to work or participate fully in family and social activities and have increased risks of infection. The incurable nature of the condition can be associated with anxiety and the overall reduction in quality of life may lead to mental health issues. Venetoclax offers an additional treatment option with the benefit of progression free survival. As an oral treatment which can be administered at home it may also allow some patients to continue work or engage more fully in family life.

Also accepted through PACE was mexiletine (Namuscla) for the treatment of symptoms of myotonia (muscle stiffness) in patients with non-dystrophic myotonic (NDM) disorders. NDM is a group of muscle disorders in which muscles are slow to relax after movement. In the PACE meeting, patient group representatives and clinicians spoke of how the main symptoms of this rare condition, including muscle stiffness, pain, weakness and fatigue, can have a significant impact on patients’ daily lives. The condition also has an emotional impact on both patients and their carers, limiting their ability to work and socialise. This formulation of mexiletine is the first medicine to be licensed for the symptomatic treatment of myotonia in patients with NDM. Mexiletine may improve symptoms which could allow some patients to be independent, relieving the anxiety and distress associated with this condition.

Bempedoic acid (Nilemdo), a medicine that can be used to lower levels of cholesterol in the blood, was not accepted because the evidence provided by the company was not strong enough to satisfy the committee that it is a good use of NHS resources.

SMC chairman Mark MacGregor said:

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

“For those with CLL, venetoclax offers another treatment option which may enable patients to continue to take part in work and family life.”

“In our PACE meeting, we heard how those with NDM disorders can struggle to carry out daily tasks, so we know our acceptance of mexiletine, the first licensed formulation of this medicine to specifically treat this condition, will be welcomed.”

“The evidence provided for bempedoic acid was not strong enough for the committee to be able to accept this medicine.”

In addition to these medicines, SMC has also accepted the following medicine through an expedited approach to minimise delay in patient access following the early phase of the COVID-19 pandemic. More details can be found on remobilisation webpage.

• Avatrombopag (Doptelet) for the treatment of severe thrombocytopenia in adults with chronic liver disease who are scheduled to undergo an invasive surgical procedure.


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