A Brief History
Since the UK NSC rejected newborn screening for SMA in 2018, the NHS has revolutionised care for children, young people and adults who have Spinal Muscular Atrophy (SMA) by securing and funding access to three ground-breaking treatments. This has been life-saving for babies who have the most severe SMA, who previously would have died before the age of 2 years. Children affected by this progressively muscle weakening condition have gained strength, movement and better health after treatment. Symptoms have stabilised for many adults, with some seeing improvements.
Our Calls To Action
It is unethical to delay the introduction of SMA newborn screening to the UK. We recognise that efforts are underway to improve how decisions are made on newborn screening in the UK. The UK National Screening Committee’s (UK NSC) high profile review of SMA newborn screening is critical to translating this ambition into meaningful progress. We are calling for three urgent changes to the UK NSC’s approach to SMA:
- Timescales – we ask the UK NSC to commit to complete its review of newborn bloodspot screening for SMA at the earliest opportunity, with clarity on the key evaluation milestones needed to reach that deadline.
- Engagement – we ask the UK NSC to take a more transparent and collaborative approach to engagement with the SMA expert clinical and patient community.
Evidence – We ask the UK NSC to adopt a streamlined and pragmatic approach to evidence assessment for SMA, to reduce the chance of further unnecessary delays.
The UK SMA Newborn Screening Alliance is working hard on a number of fronts to try to ensure a rapid positive decision on newborn screening for SMA. We are delighted that the review is finally underway and have outlined some of our work below:
• Engagement with the UK NSC: we have been engaging with the UK National Screening Committee (UK NSC) on its review of SMA. In liaison with our steering group, we are collating evidence to share with the team from Sheffield School of Health and Related Research, which is conducting a review of the evidence available to support newborn screening for SMA.
• Raising awareness and understanding: we have been speaking to a wide range of parliamentarians and policy-maker stakeholders to raise awareness of SMA and the strength of the case for newborn screening. We have also developed briefings and materials to help demystify some aspects of the debate. If you would like to know how you can help us with some of our parliamentary engagement, please click on the link below.