February 2022 decisions news release

Advice on six new medicines for use in conditions including a rare lung cancer caused by occupational exposure to asbestos and also for a severely disabling muscle wasting disease has today (Monday 7 February) been published by the Scottish Medicines Consortium (SMC).

Nivolumab (Opdivo) was accepted for the treatment of advanced malignant pleural mesothelioma, a type of lung cancer caused by exposure to asbestos that affects the thin membrane that lines the lungs. Nivolumab 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 spoke of the importance of the survival benefit, of four months on average, and longer duration of response compared with conventional chemotherapy, potentially allowing patients valuable additional time with friends and family. Nivolumab is an immunotherapy – a treatment that stimulates the immune system to fight the cancer – and is administered in combination with another medicine, ipilimumab.

Risdiplam (Evrysdi) was accepted for the treatment of spinal muscular atrophy (SMA), a rare muscle wasting disease caused by a faulty gene. SMA is a severely disabling condition that causes weakness affecting all the muscles in the body. Treatment options are limited. Risdiplam is the first oral treatment so offers another treatment option for this devastating condition. In the PACE meeting, patient groups and clinicians highlighted that, in their experience, improvements with risdiplam may allow patients to be less dependent on family and carers in many ways, including needing assistance with activities of daily living and self-caring and for support during the night.

Cannabidiol (Epidyolex) was accepted for the treatment of patients aged two years and over with seizures associated with tuberous sclerosis complex, a rare genetic condition. Through PACE, patient groups and clinicians told of how patients usually require round the clock care and supervision which impacts on all aspects of life including family, social and education. Cannabidiol is taken orally in combination with a patient’s current treatment and may reduce the number of seizures per day. Any improvements in seizure control could have a substantial impact on patients and carers, with the potential for increased participation in family and social activities. Cannabidiol offers a further option to tailor treatment for patients.

Also accepted for the treatment of epileptic seizures was cenobamate (Ontozry). Cenobamate is used as an add-on to other epilepsy medicines in patients with seizures that are not controlled despite having tried at least two other treatments, also known as drug-resistant epilepsy. Cenobamate is taken orally and addresses the high unmet need in patients who are considered to have drug-resistant epilepsy. A patient group submission highlighted that cenobamate may improve seizure control, allowing patients to live safer, healthier and more independent lives.

Pemigatinib (Pemazyre) was accepted for the treatment of advanced cholangiocarcinoma, a cancer of the bile ducts in patients with a specific gene mutation. This is an aggressive cancer with a poor prognosis with very limited treatment options. The evidence for pemigatinib is still emerging but PACE participants described their experience of using this treatment, noting that it rapidly improved symptom control and delayed tumour progression. They stressed that it provides an additional treatment option to chemotherapy which offers a huge benefit to those patients not fit enough for this kind of treatment. Pemigatinib is taken orally, offering convenience over chemotherapy.

Enzalutamide (Xtandi) was accepted for the treatment of prostate cancer that has spread to other parts of the body and is used alongside androgen deprivation therapy (hormone treatment). PACE participants highlighted that this type of cancer is incurable and patients often experience difficulties with mobility and carrying out daily activities. Enzalutamide provides an additional oral treatment option that can slow disease progression and increase survival.

SMC chairman Mark MacGregor said:

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

“Mesothelioma is an aggressive cancer with a poor prognosis and limited treatment options. Nivolumab has the potential to improve the quality of life for patients living with this advanced cancer and may give patients valuable extra time with friends and family. ”

“Spinal muscular atrophy is a devastating condition both for patients and their families. As the first oral treatment, risdiplam is another more convenient treatment option for some patients.”

“For people living with tuberous sclerosis complex, cannabidiol may reduce the number of seizures experienced each day, leading to marked improvements in quality of life for both patients and their carers.”

“Cenobamate provides an additional option for patients with drug-resistant epilepsy, a condition that can be difficult to manage.”

“Cholangiocarcinoma is a rare form of bile duct cancer with very few effective treatment options. Pemigatinib offers a more personalised treatment and can be taken at home giving patients more quality time to spend with family - a benefit that was strongly communicated by the PACE participants.”

“For some patients living with incurable prostate cancer, enzalutamide can slow disease progression improve quality of life and potentially allow patients to live longer.”


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