June 2019 decisions news release

The Scottish Medicines Consortium (SMC), which advises on newly licensed medicines for use by NHSScotland, has today (Monday 10 June) published advice accepting five new medicines.

Patisiran (Onpattro) was accepted for the treatment of hereditary transthyretin (hATTR) amyloidosis following consideration through SMC’s Patient and Clinician Engagement (PACE) process, which is used for medicine to treat end of life and very rare conditions. Hereditary transthyretin amyloidosis is a very rare progressive genetic disease in which abnormal proteins called amyloid builds up in tissues around the body including the nerves and the heart. Through PACE, patients groups and clinicians emphasised the heavy symptom burden of the disease, with neuropathy (nerve disorder) symptoms including loss of sensation, weakness and muscle wasting. In addition, most patients experience bladder problems, diarrhoea, incontinence and vomiting. The severe and worsening disability experienced by patients affects all areas of life for them, their families and carers. Patisiran is the first medicine available to treat the underlying defects of amyloidosis and has the potential to halt or slow the progression of the disease, and in some cases may improve symptoms, leading to a better quality of life.

Brigatinib (Alunbrig) was accepted for the treatment of a rare, advanced form of non-small cell lung cancer (NSCLC) in patients who have not responded to another cancer medicine called crizotinib. It is only used if the NSCLC is ‘ALK-positive’, which means the cancer cells have defects in the gene responsible for a protein called ALK (anaplastic lymphoma kinase). Through PACE, patient groups and clinicians explained that this type of lung cancer often affects younger, fitter, non-smoking patients. As it usually presents at a late stage when it has already spread to the brain, it can have a profound impact on quality of life and symptoms can be particularly difficult to manage. There are limited treatment options for this patient group. Brigatinib may extend the time before the cancer returns and improve overall survival.

Durvalumab (Imfinzi) was accepted for the maintenance treatment of locally advanced non-small cell lung cancer (NSCLC). It is used in adult patients with advanced NSCLC that cannot be removed by surgery and did not get worse after treatment with chemoradiation therapy (where chemotherapy and radiotherapy are given at the same time). Durvalumab, which is an immunotherapy, is used when the tumour produces a protein known as PD-L1. Patient groups and clinicians highlighted through PACE that NSCLC has a poor prognosis and, following chemoradiation therapy, patients are currently monitored for signs of disease progression and are not given active treatment until the disease progresses. Administration of durvalumab maintenance therapy may prevent or delay further growth and spread of cancer, and may increase overall survival.

Nivolumab (Opdivo) was accepted in combination with another medicine, ipilimumab, for the treatment of advanced renal cell carcinoma, a form of kidney cancer. Patient group submissions noted that renal cancer was often diagnosed incidentally, when patients were seeking help for other conditions. There are currently limited treatment options for this condition. Nivolumab with ipilimumab was shown to improve progression free survival times and overall response rates compared to other current treatment options.

Benralizumab (Fasenra) was accepted for the treatment of severe eosinophilic asthma (a type of asthma characterised by an excess of inflammatory cells known as eosinophils) which is not well controlled with other treatments. Severe eosinophilic asthma does not respond to standard treatment and requires more intensive therapies to control symptoms to prevent attacks, hospitalisations and deaths. Benralizumab provides another treatment option and has been shown to reduce asthma attacks and can reduce the amount of other medicines required to treat the condition.

SMC Chairman Dr Alan MacDonald said:

“Our committee members are pleased to be able to accept these five new medicines for use by NHSScotland.”

“Patients with hereditary transthyretin amyloidosis have a poor prognosis and heavy symptom burden that affects every aspect of their lives and those of their families and carers. We know from the evidence given through our PACE meeting that our decision on patisiran will be welcomed.”

“For patients with ALK-positive NSCLC, brigatinib offers the opportunity of more good quality time with family and friends before the cancer returns. For those with PD-L1-positive NSCLC, durvalumab maintenance therapy can help delay the spread of the cancer and offers the opportunity of improved survival.”

“Our decision on nivolumab for the treatment of advanced renal cell carcinoma means patients may have longer without the progression of the cancer compared to other currently available treatments.”

“Benralizumab will help those with severe asthma better manage their condition and hopefully reduce the volume of other medications they require.”

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