UK NSC minutes June 2023

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Top Highlights

• In July 2021, the UK NSC held a stakeholder workshop where stakeholders were given the opportunity to contribute to the UK NSC’s approach to examining the evidence for SMA given the evolving landscape. Following this, scoping work for a decision analytic model and cost effectiveness evaluation of newborn screening for SMA was commissioned by the UK NSC in 2022 and was awarded to the School of Health and Related Research (ScHARR) at the University of Sheffield.

• flexible cost effectiveness modelling study for the UK screening context has been proposed, to assess the uncertainties and unanswered questions from the 2 objectives.

• use in presymptomatic SMA, and the outcomes of the other models summarised in the confidential ScHARR report

• provide reasonable grounds for more formally exploring a UK newborn screening programme for SMA

• Members of the UK NSC supported the need to have a new independent model and were happy to agree to this. The committee also supported the call to start planning for ISE work for SMA but emphasised that this was not a recommendation to introduce screening. Rather, it was a pragmatic and innovative way in which the new model could help define key issues for the ISE and the ISE could then be used to collect data to feed into the model and test its assumptions

• inform the evidence on which a UK NSC recommendation on newborn screening for SMA will be made.