Risdiplam for treating spinal muscular atrophy in children and adults – Final appraisal document

In DRAFT guidance, NICE recommends risdiplam for treating 5q spinal muscular atrophy in people aged ≥2 months with a clinical diagnosis of SMA types 1, 2 or 3 or with pre-symptomatic SMA and 1 to 4 SMN2 copies, only if the conditions of the managed access agreement are followed.

SPS commentary:

The managed access agreement is a special arrangement between NHS England and NHS Improvement and Roche, which will allow people to access treatment with risdiplam while more data is collected to address the uncertainties identified by the independent NICE committee. After this NICE will decide whether or not to recommend it for routine use on the NHS and update the guidance.

The committee could not recommend risdiplam for routine commissioning at this time because the cost-effectiveness estimates were substantially higher than the range normally considered a cost-effective use of NHS resources. In the case of type 1 spinal muscular atrophy (SMA), this was despite agreeing that risdiplam could be considered a life-extending treatment at the end of life.

NICE already recommends nusinersen for some people with types 1,2 and 3 SMA and pre-symptomatic SMA as part of a managed access agreement. NICE has also published guidance which recommends onasemnogene abeparvovec for some babies aged up to 12 months with type 1 SMA and for treating pre-symptomatic SMA.

National Institute for Health and Care Excellence