NICE update: treatment for SMA patients gets go-ahead

NICE published nine technology appraisals, one public health guideline and one diagnostics guidance item this summer.

Under guidance TA588, Nusinersen for treating spinal muscular atrophy, more children with the rare genetic disorder spinal muscular atrophy (SMA) can be treated. This followed a proposal to extend the terms of the managed access agreement between NHS England and Biogen for funding it.

SMA affects the nerves in the spinal cord controlling movement, causing muscle weakness, progressive movement loss and difficulty breathing and swallowing. Those with the most severe forms usually die before the age of two. Without nusinersen, the condition is managed with supportive care to minimise the impact of disability, tackle complications and improve quality of life.

A further technology appraisal, TA593, recommends ribociclib used with fulvestrant as an option for people with hormone receptor-positive, human epidermal growth factor receptor 2 (HER2)-negative, locally advanced or metastatic breast cancer who have had previous endocrine therapy.

The positive recommendation follows an improved patient access scheme by the company as part of a commercial arrangement. The appraisal committee recognised that ribociclib with fulvestrant has the potential to be cost-effective, and recommended it for use in the Cancer Drugs Fund. This will allow more evidence to be collected to address the uncertainties around overall survival and cost-effectiveness.

Elsewhere, guideline NG135, Alcohol interventions in secondary and further education, replaces NICE public health guideline PH7 (November 2007). The interventions aim to prevent and reduce alcohol use among those aged 11 to 18. It also covers people aged 11 to 25 with special educational needs or disabilities in full-time education. Costs associated with implementing the guideline are not expected to be significant.