September 2022 decisions news release
The Scottish Medicines Consortium (SMC), which advises on newly-licensed medicines for use by NHSScotland, has today (Monday 12 September) published advice on seven new medicines.
Delta-9-tetrahydrocannabinol and cannabidiol (Sativex®) was accepted as treatment for symptom improvement in some adults with spasticity due to multiple sclerosis (MS). MS can cause a wide range of involuntary muscle spasms, particularly in the legs. The committee heard from MS patient groups about the debilitating effect that muscle spasms can have on their day-to-day lives, impairing mobility, increasing the risk of falls, affecting ability to self-care and aggravating fatigue and depression. Sativex is an oral spray treatment taken in combination with a patient’s current treatment.
Nivolumab (Opdivo®), given in combination with chemotherapy, was accepted as a treatment for some adults with gastro oesophageal junction cancer – the area where your food pipe joins the stomach. It was considered through SMC’s Patient and Clinician Engagement (PACE) process, which is used for medicines for end of life and rare conditions. The addition of nivolumab to chemotherapy may increase survival, allowing patients to spend more time with family and loved ones.
The committee accepted apalutamide (Erleada®) as a treatment for some patients with prostate cancer that has spread to other parts of the body. Apalutamide, given in combination with androgen deprivation therapy, provides an additional oral treatment option that can slow disease progression and increase survival.
The committee accepted imlifidase (Idefirix®), a treatment for highly sensitised adult kidney transplant patients to improve their chances of avoiding organ rejection after transplant. It is the first medicine licensed for desensitisation treatment of patients with positive match against an available deceased donor. Imlifidase was also considered through the PACE process. PACE participants spoke of the burden of dialysis appointments, chronic fatigue and the impact on their quality of life and mental health. Imlifidase may enable some patients to receive a deceased-donor kidney transplant where this would otherwise not be possible. After a successful kidney transplant, a patient and their family would enjoy life-changing benefits.
Tofacitinib (Xeljanz®) was accepted for the treatment of adults with ankylosing spondylitis (AS), a chronic inflammatory rheumatic disease that affects the spine and pelvis. In patients with AS, joint mobility generally worsens over time and tofacitinib offers an alternative oral treatment option that may reduce associated symptoms and improve quality of life.
An initial assessment report has been published for velmanase alfa (Lamzede®), a treatment for a rare genetic condition called alpha-mannosidosis. The condition is caused by a faulty gene. Children who develop the disease can suffer a range of issues including skeletal abnormalities, recurrent infections and muscle weakness. Velmanase alfa has been assessed through the ultra-orphan pathway for medicines aimed at extremely rare conditions. It will be available for three years while further information is gathered. Following this, SMC will review the evidence and make a decision on routine availability in NHSScotland.
The committee was unable to accept zanubrutinib (Brukinsa®) for the treatment of Waldenstrom’s macroglobulinemia, as there was uncertainty around its cost effectiveness. The evidence provided by the company was not strong enough to satisfy the committee that it offers value for money to NHSScotland.
SMC chairman Mark MacGregor said:
“I am pleased we were able to accept these medicines for use by NHSScotland.
“We heard powerful testimony from MS groups of the devastating effects of spasticity on patients with multiple sclerosis. Delta-9-tetrahydrocannabinol and cannabidiol will provide a treatment option to improve quality of life for patients, where other treatments are not working well enough.
“Nivolumab may increase survival in patients with advanced gastro oesophageal cancer and we know that even small extensions in duration of life are of considerable importance to patients and families.
“A metastatic prostate cancer diagnosis brings a heavy burden on patients, and symptoms get worse as disease progresses. Apalutamide will offer another useful alternative oral treatment in helping to slow disease progression and extend life.
“When considering imlifidase we heard through the PACE meeting from patients who are aware that they have limited opportunities to safely receive a kidney transplant and that their health and prognosis on dialysis can deteriorate to the point where they may no longer be fit to have a transplant. Imlifidase will increase their chances to receive a life-saving transplant.
“Whilst chronic back pain is the predominant symptom of ankylosing spondylitis, the knock-on effects of pain and fatigue can have a major impact on wellbeing. Tofacitinib will provide a treatment option that may help improve this.
"Patients will also gain access to velmanase alpha while the company gathers additional evidence to inform a decision in three years. Alpha-mannosidosis is an extremely rare and debilitating condition, velmanase alpha will provide a treatment that may offer some relief from the symptoms. The costs were extremely high relative to the identified benefits and this additional time will give the company the opportunity to provide greater certainty around the benefits and impacts on resource use.
“We were unable to accept zanubrutinib as the evidence provided by the company on the benefits of using this medicine was not strong enough to justify its cost.”