Detailed Academic Paper on Cost-Effectiveness of Newborn Screening for SMA in England is Now Available

As part of its decision-making process, the UK National Screening Committee (UK NSC) considers economic modelling to be important for rare diseases, given the limitations on available evidence.

The UK SMA Newborn Screening Alliance is delighted that the UK NSC is open to considering existing economic models on the cost effectiveness of newborn screening for SMA.

SMA Alliance Chair, Laurent Servais, has worked with others on a model that evaluates the cost effectiveness of newborn screening for SMA in England and Wales, versus not introducing newborn screening for SMA.

The model has found, as set out in detail in this pre-print of the academic paper, that:

• The introduction of newborn screening for SMA in England and Wales is estimated to identify approximately 96% of cases of SMA in infants per year.
• Introducing newborn screening would be less costly and more effective than not introducing it.
• Introducing newborn screening for SMA would result in savings of over £62million over the course of the lifetime of the cohort of newborns diagnosed with SMA each year.
• There would be an estimated gain in quality-adjusted life years (QALYs) of 529 years over the lifetime of this cohort.

Looking at existing models, rather than creating a new model from scratch, will help streamline UK NSC timescales as far as possible, making it easier for a quicker decision to be taken to add SMA to the national heel-prick test.

Although any ‘third party model’ will need to be quality assured, efforts can be made to ensure that this process is not unduly long. For example, the third party model collaborators should be engaged with at an early stage to answer questions about the model and expedite the QA process.

The Alliance is keen to facilitate this engagement and ensure that deliberations on newborn screening for SMA in the UK are efficient and a decision is made as swiftly as possible.