Notes | Project documents | Nusinersen and risdiplam for treating spinal muscular atrophy (review of TA588 and TA755) [ID6195] | Guidance

Following the committee meeting on 11 November, NICE is pausing this appraisal. This is because while good progress was made in resolving some key issues, the committee has identified some additional analysis that it needs to help it make recommendations. This analysis relates to the economic modelling in 3 broad areas: patient health-related quality of life values, healthcare resource use and carer health-related quality of life. The aim of seeking additional analysis in each of these areas is to make sure that the economic modelling adequately captures the benefits of the treatments that were described by the patient and clinical experts and seen in the clinical data. As this analysis is of a technical nature and related to the company modelling, NICE considers that the companies are best placed to provide it. Once this analysis is available and has been reviewed, a second public committee meeting will be scheduled to discuss it. NICE will not be releasing a draft guidance document until after this second committee meeting. It is likely that the second committee discussion will be held in early 2026.