New model on the cost effectiveness of newborn screening for SMA in England

A new model has been developed to explore the cost effectiveness of screening for spinal muscular atrophy (SMA) in England. It has found that newborn screening can give babies with SMA a healthier future by enabling treatment before symptom onset in the vast majority of cases. This approach will generate savings of over £67million across the lifetime of all of the babies identified in one year.

The findings generated by the model were presented this week at ISPOR, the leading European conference for health economics and outcomes research.

The UK SMA Newborn Screening Alliance welcomes the development of this model and hopes that the UK National Screening Committee will ensure that it is reviewed and incorporated into the official newborn screening assessment process for SMA. This new evidence should help to ensure that a robust but rapid decision can be made about adding SMA to the national newborn screening programme in the UK.

More about the model:

A model is built using data and assumptions to try to predict real-life outcomes. In this case, the model has been designed to understand the impact of introducing newborn screening in England, versus not doing so. The results have indicated that newborn screening is less costly and more effective compared to not having newborn screening. The model predicted that newborn screening for SMA would lead to the testing of 585,195 newborns and the identification of approximately 59 with SMA.

Importantly, the model identified a cost saving of over £67million over the lifetime of the newborn cohort of 59 children identified. Timely diagnosis via newborn screening would enable the majority of these babies to be treated prior to symptom onset, which would lead to better outcomes from treatment and a reduction in direct and indirect costs associated with the disease.

The model also showed that across the lifetime of the 59 identified babies with SMA in one year, there would be an estimated gain of 567 QALYs. A QALY is a quality-adjusted life-year. It is intended to be a measure of the value of health outcomes, combining the quality of life and length of life after treatment.

The poster presented at ISPOR, which contains further information on the model, can be viewed here.