Posted by Michael Wonder on 20 Nov 2021
NICE draft guidance recommends new treatment for spinal muscular atrophy as part of a managed access agreement
19 November 2021 - Around 1,500 people in England with the rare genetic disorder spinal muscular atrophy are set to benefit from a new treatment after NICE today published draft guidance recommending risdiplam (Evrysdi; Roche) as part of a managed access agreement.
The managed access agreement is a special arrangement between NHS England and NHS Improvement and Roche. It 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. It will be available through the managed access agreement until then.
