Real World Evidence on the Effectiveness of Nusinersen within the National Program to Treat Spinal Muscular Atrophy in Poland

Background: Spinal muscular atrophy (SMA) is a debilitating neuromuscular disease resulting in children’s mortality and disability. Nusinersen is available to all SMA patients in Poland since 2019. Aim: To compare mortality or disease progression to mechanical ventilation in two patient cohorts before and after the program’s introduction. Additionally, to describe the patient population treated with nusinersen and costs incurred by the public payer. Methods: We used the National Health Fund (NHF) database to identify patients born in either 2014 or 2019, who received at least two health services with an ICD10 G12 diagnosis. Outcomes were time to event: death or first mechanical ventilation. We identified all benefits received by nusinersen-treated patients, between 1 January 2019 and 31 May 2022. Results: Children with SMA born in 2019 had significantly lower mortality in the first years of their lives than children born in 2014. Approximately 875 patients (all age groups) were treated with nusinersen in the analysis period. The cost of causal drugs in this period amounted to €51.4 million. The cost of healthcare benefits amounted to €14.9 million. Conclusions: The drug program to treat SMA improved patient care in Poland. The NHF database was a reliable source to monitor resource-intensive therapies’ costs, demography, and selected patient outcomes.