Presentation (CTI)

OBJECTIVES: The European Medicines Agency recently approved a novel, high dose (HD) nusinersen regimen (50/28 mg) for the treatment of 5q spinal muscular atrophy (SMA). Evidence from the registrational trial DEVOTE Part B infantile-onset, suggests that HD nusinersen may reduce hospital admissions due to serious adverse events (SAEs) and also reduce serious respiratory events (SREs) compared to the standard dose (SD) regimen (12/12 mg). Additionally, HD nusinersen offers a reduced administration frequency during first year of treatment. This analysis aims to estimate the potential reduction in healthcare resource utilization and hospital costs associated with HD nusinersen from the Italian National Health Service (NHS) perspective.
METHODS: A 1-year economic analysis was conducted on a hypothetical cohort of 100 SMA infantile-onset patients. Clinical inputs, SAEs/SREs and length-of-stay, were derived from DEVOTE Part B trial (infantile-onset). Resource consumption was valued using Italian NHS reference costs, considering full and ER hospital admission tariff for SAEs/SREs, respectively. The analysis compared total hospital and administration costs during first year of treatment between HD and SD nusinersen regimens.
RESULTS: Compared to SD, the HD nusinersen regimen was associated with fewer SAE-related hospitalizations (30.4 vs 78.4; difference: -48.0), cumulative length-of-stay (2,579 days vs 933 days; difference: -1,646), and fewer SRE cases (93.6 vs 134.4; difference: -40.8). HD nusinersen reduced the number of administrations by 200 (400 vs 600). These clinical improvements and the reduction in administrations would translate into significant cost offsets, with total hospital costs of €383 thousand for HD nusinersen vs €739 thousand for SD nusinersen, resulting in potential savings of €356 thousand (relative reduction: -48%).
CONCLUSIONS: In the Italian NHS setting, HD nusinersen has the potential to generate substantial cost savings for the treatment of infantile-onset SMA patients by reducing clinical and economic burden of hospital admissions and administration frequency, during first year of treatment.