Spinal Muscular Atrophy: Screening
The question
To ask the Secretary of State for Health and Social Care, what is the composition of the control group on the In-Service Evaluation in relation to spinal muscular atrophy screening.
Answered by Preet Kaur Gill
The Department invests in research through the National Institute for Health and Care Research (NIHR). The NIHR is funding a £4.1 million study on in-service evaluation of newborn screening (NBS) for spinal muscular atrophy (SMA), led by Professor Laurent Servais at the University of Oxford. The purpose of this study is to provide evidence of the feasibility, acceptability, efficacy, and cost effectiveness of NBS for SMA.
The study is using a type of cluster randomized trial called a ‘stepped wedge design’, where laboratories taking part in the study progressively role out the screening of newborns for SMA in phases. Laboratories act as the ‘control group’ before they start to screen newborns for SMA. All groups eventually receive the screening. By comparing the outcomes of babies diagnosed before entering the study with those diagnosed through screening later in the study, researchers can assess whether NBS for SMA improves outcomes such as time to treatment, motor development, and cost-effectiveness.
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