July 2022 decisions news release

The Scottish Medicines Consortium (SMC), which advises on newly licensed medicines for use by NHSScotland, has today (Monday July 11) published advice on five new medicines.

Crizanlizumab was accepted for use in patients with sickle cell disease on an interim basis subject to ongoing evaluation and future reassessment. Sickle cell disease is a rare genetic condition where the red blood cells become sickle shaped. This can block the flow of blood in blood vessels, causing painful episodes known as vaso-occlusive crises. 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. PACE participants highlighted that these painful crises can be frequent and unpredictable, with the most severe episodes requiring hospitalisation for treatment and analgesia. Treatment options are extremely limited and crizanlizumab may offer a step forward in managing the condition and reducing the number of crises. The clinical evidence in the company submission had some limitations and the committee therefore accepted crizanlizumab on an interim basis subject to ongoing evaluation. SMC will consider further evidence on its effectiveness once this is available. Further information on interim acceptance is available on our website.

An initial assessment report has been published for odevixibat (Bylvay), which can be used to treat progressive familial intrahepatic cholestasis (PFIC). This medicine has been assessed through the ultra-orphan pathway for medicines that treat very rare conditions. PFIC is a rare group of conditions that cause bile acids to build up in the liver causing damage, with many patients progressing to end stage liver failure and needing a liver transplant. The severe itchiness caused by the condition has a big impact on quality of life, leading to skin damage, sleep deprivation, irritability, poor attention and difficulties at school. The condition also has considerable impact on carers and siblings. Clinical trials showed that odevixibat may reduce serum bile acids and itchiness leading to an improved quality of life. However there is still uncertainty about whether it delays the need for liver transplant, and despite the confidential discount offered by the company, the cost in relation to the health benefits of odevixibat remains high. 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. Odevixibat 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.

Solriamfetol was accepted for the treatment of narcolepsy, a rare, lifelong neurological disorder that causes involuntary napping, excessive need for sleep, difficulty sustaining attention and brief involuntary sleep episodes. Through the PACE process, SMC committee heard that narcolepsy has a daily chronic disabling impact on people’s relationships and work and home life. Solriamfetol may help improve wakefulness, most likely where current treatments been unsuccessful or are unsuitable for the individual. Less sleepiness could result in improved quality of life for some patients.

Delafloxacin, an antibiotic, was accepted for the treatment of acute (short-term) bacterial skin infections such as cellulitis, skin abscesses and wound infections. It can be used when other commonly prescribed antibiotics have not worked or are not considered suitable.

Pegcetacoplan was accepted for the treatment of adults with paroxysmal nocturnal haemoglobinuria (PNH), a rare and potentially life-threatening condition where part of the body’s immune system is overactive and breaks down too many red blood cells. This can lead to anaemia, tiredness, difficulty in functioning and swallowing, pain and blood clots. The medicine can be used for those who are still anaemic despite receiving treatment with another type of medicine used to treat PNH.

SMC Chairman Mark MacGregor said: “The committee is pleased to be able to accept these medicines for use by NHSScotland."

“Sickle cell disease is a condition that has a severe impact on patients’ health and well-being and affects all aspects of daily activities including education or work. We are pleased to be able to accept crizanlizumab as it may reduce the frequency of crises, relieving the severe pain and distress and the associated risks. It may improve the experience of care and wellbeing of patients who experience fewer unpredictable pain crises, allowing them to participate more fully in daily and social activities. This in turn may improve their quality of life, mental health and independence."

“Odevixibat (Bylvay) will now move to the next stage of the ultra-orphan pathway. This will allow patients with PFIC 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.”

“Solriamfetol offers another treatment option to help people with narcolepsy maximise their lives on a day to day basis.

“Antibiotic resistance is an ongoing concern and the availability of another antibiotic, delafloxacin, is to be welcomed."

“For those with paroxysmal nocturnal haemoglobinuria (PNH), pegcetacoplan, offers another treatment option for a very serious condition and the potential for improved levels of wellbeing.”


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