NHS Scotland's June 2019 Funding Decisions

Nicky Broyd

June 11, 2019

The Scottish Medicines Consortium (SMC) has this month approved five new drugs - for hereditary transthyretin amyloidosis, non-small cell lung cancer, renal cell carcinoma, and eosinophilic asthma.

Hereditary Transthyretin Amyloidosis

Patisiran (Onpattro, Alnylam) was accepted for the treatment of the rare genetic disease, hereditary transthyretin (hATTR) amyloidosis. This followed consideration via the Patient and Clinician Engagement (PACE) process for rare conditions and end of life care.

In hATTR abnormal proteins build up in body tissues causing progressively worsening symptoms including neuropathy, weakness, muscle wasting, bladder problems, diarrhoea, incontinence, and vomiting.

Patisiran may halt or slow disease progression and improve symptoms. It is the first medicine made available to treat the underlying defects of amyloidosis.

SMC Chairman Dr Alan MacDonald said: "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."

Non-small Cell Lung Cancer

Two drugs were approved for non-small cell lung cancer (NSCLC).

Brigatinib (Alunbrig, Takeda) was approved for ALK (anaplastic lymphoma kinase)-positive advanced NSCLC where patients haven't responded to crizotinib (Xalkori, Pfizer).

Representations through the PACE process from doctors and patient groups stressed how the condition often affects younger, fitter, non-smoking patients.

It usually presents at a late stage when cancer has spread to the brain, and its effects have a severe impact on quality of life, with difficult to manage symptoms and few treatment options.

Brigatinib may improve overall survival and may extend the time before cancer returns.

Durvalumab (Imfinzi, AstraZeneca) immunotherapy treatment was accepted for maintenance treatment of advanced NSCLC in adults where the cancer cannot be removed by surgery and that did not get worse following chemoradiation. It is used when the tumour produces a protein called programmed death ligand-1 (PD-L1).

PACE representations stressed the poor prognosis with this form of cancer.

Durvalumab maintenance therapy may increase overall survival and may prevent or delay further cancer growth and spread.

Dr MacDonald said: "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."

Renal Cell Carcinoma

Nivolumab (Opdivo, Bristol-Myers Squibb) was accepted in combination with ipilimumab (Yervoy, Bristol-Myers Squibb) for the treatment of advanced renal cell carcinoma.

Patient group representations highlighted how renal cancer was often diagnosed during investigations for other conditions and has limited treatment options.

Nivolumab with ipilimumab has been shown to improve progression-free survival times and better overall response rates compared with other current treatments.

Dr MacDonald said: "Our decision on nivolumab for the treatment of advanced renal cell carcinoma means patients may have longer without the progression of the cancer compared with other currently available treatments."

Eosinophilic Asthma

Benralizumab (Fasenra, AstraZeneca) was accepted for the treatment of severe eosinophilic asthma not well managed using other treatments. Severe eosinophilic asthma does not respond to current standard treatments and requires more intensive therapy to manage symptoms, and to prevent asthma attacks, hospital admissions, and deaths.

There's evidence benralizumab may reduce attacks as well as the need for other medications to treat the condition.

Dr MacDonald said: "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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