The Scottish Medicines Consortium (SMC), has today (Monday 11 June) published advice accepting six new medicines for use by NHSScotland. Five of the medicines were accepted through SMC’s Patient and Clinician Engagement (PACE) process, which is used to consider medicines for treating end of life and very rare conditions.
Everolimus (Votubia) was accepted for the treatment of epilepsy in patients with tuberous sclerosis complex (TSC). TSC is a rare genetic disorder affecting both children and adults that causes mainly non-cancerous (benign) tumours to develop in different parts of the body, including the brain. The majority of patients have TSC related epilepsy and experience frequent seizures. Through PACE, patient groups and clinicians spoke of how existing epilepsy medication is often unable to control these seizures which means that patients live with the constant fear, anxiety and exhaustion of having to manage their condition. Everolimus can reduce the frequency of seizures, offering patients a better quality of life.
Inotuzumab ozogamicin (Besponsa) was accepted for the treatment of acute lymphoblastic leukaemia (ALL). The PACE meeting participants reported that ALL is a rare, very aggressive, rapidly progressing form of leukaemia with a particularly poor prognosis and the only possibility of long term survival is a stem cell transplant. Chemotherapy is often ineffective for these patients and the majority are unable to go on to receive a transplant. Inotuzumab ozogamicin can improve rates of remission and increase a patient’s chance of receiving a transplant. As it can be delivered without requiring a stay in hospital and is better tolerated than other treatments, it also offers the opportunity to improve quality of life.
The committee also accepted midostaurin (Rydapt) for adult patients with newly diagnosed acute myeloid leukaemia (AML). PACE participants told the meeting that AML is a rapidly progressing form of leukaemia, with approximately half of patients being diagnosed following emergency presentation, and approximately 80% commencing treatment within a week of diagnosis. When midostaurin is added to current treatment it can improve survival and reduce the risk of disease relapse without substantial side-effects.
Crizotinib (Xalkori) was accepted for the treatment of a rare subtype of non-small cell lung cancer (NSCLC). Evidence presented at the PACE meeting told of how patients are often diagnosed at an advanced stage of the disease and have short life expectancy. Crizotinib is the first targeted therapy for this group of patients and is associated with a high response rate. Evidence on use of crizotinib in a similar subtype of NSCLC indicates it is likely to result in an extended period of progression free and overall survival compared to chemotherapy treatment. Crizotinib is administered orally and would allow patients to be treated at home, reducing the need for outpatient visits.
Also accepted through PACE was telotristat ethyl (Xermelo), which can be used to treat severe diarrhoea associated with a rare condition called carcinoid syndrome which can occur with a certain type of tumour. PACE participants told the meeting how this condition can be extremely debilitating and distressing resulting in a poor quality of life. Patients may have to give up work and are often reluctant to travel away from home. Telotristat ethyl can increase symptom control thereby improving a patient’s quality of life. Also, as an oral therapy it can also be easily administered at home.
The committee also accepted guselkumab (Tremfya) for the treatment of severe plaque psoriasis in adults. Guselkumab offers another treatment option for those patients who have failed to respond to standard therapies.
SMC Chairman Dr Alan MacDonald said:
“The committee is pleased to be able to accept these medicines for use by NHSScotland.”
“From the evidence we heard through PACE, we know our decision on everolimus will be welcomed as it should help patients, their families and carers to better manage what can be a very distressing condition.”
“For those with acute lymphoblastic leukaemia, inotuzumab ozogamicin offers not just an improved remission rate but also the bridge to transplant and a potential cure. Patients with acute myeloid leukaemia will hopefully benefit from our decision to accept midostaurin, which can offer improvement in survival.”
“As the first available targeted therapy, crizotinib offers those with a rare type of NSCLC the chance of a more effective treatment.”
“Participants in our PACE meeting made clear how welcome telotristat ethyl will be to those suffering from severe diarrhoea associated with carcinoid syndrome. This treatment will hopefully provide them with the opportunity for a better quality of life.”
“Guselkumab offers another useful treatment for those suffering from psoriasis who have not responded to conventional treatments.”
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