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Screening for Spinal Muscular Atrophy Enhances Nusinersen Cost-Effectiveness

January 22, 2021

For nusinersen to be cost-effective without newborn screening for infantile-onset spinal muscular atrophy, the cost of the drug would need to be reduced substantially, according to a study published in The Journal of Pediatrics. 

“Universal newborn screening for spinal muscular atrophy provides improved economic value for payers and patients when nusinersen is available,” wrote researchers from Weill Cornell Medical College and the University of Utah School of Medicine. 

Their cost-effectiveness study combined data from clinical trials with US epidemiologic and cost data. 

The incremental cost-effectiveness ratio (ICER) for nusinersen with screening was $330,558 per event-free life year saved compared with no screening and no treatment, according to the study. The incremental cost-effectiveness ratio for nusinersen without screening was $508,481 per event-free life year saved. 

At a $500,000 willingness-to-pay threshold, nusinersen with screening was the preferred strategy 93% of the time, probabilistic sensitivity analysis showed. 

“For nusinersen with screening to be cost-effective at a willingness-to-pay threshold of $50,000 per event-free life year saved, the price would need to be $23,361 per dose, less than one-fifth its current price of $125,000,” researchers advised. 

The authors noted that early data from the NURTURE trial showed an 85.7% improvement in expected life years saved compared with base results in their study. 

Jolynn Tumolo 


Jalali A, Rothwell E, Botkin JR, Anderson RA, Butterfield RJ, Nelson RE. Cost-Effectiveness of Nusinersen and Universal Newborn Screening for Spinal Muscular Atrophy. J Pediatr. 2020;227:274-280.e2. doi:10.1016/j.jpeds.2020.07.033

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