REAL-WORLD DATA OF PATIENT CHARACTERISTICS, HEALTHCARE RESOURCE USE AND COSTS AMONG RISDIPLAM-TREATED PAEDIATRIC PATIENTS WITH SPINAL MUSCULAR ATROPHY IN DENMARK
Author(s)
Peter Bo Poulsen, MSc, PhD1, Agnete Pade, MSc2, Jens Olsen, MSc2, Louise Buciek, MD3, Maya Friis Kjaergaard, MSc, PhD1, Alfred Peter Born, MD, PhD3.
1Roche Pharmaceuticals a/s, Copenhagen, Denmark, 2EY Parthenon P/S, Frederiksberg, Denmark, 3Department of Child- and Adolescent Medicine, Copenhagen University Hospital, Copenhagen, Denmark.
1Roche Pharmaceuticals a/s, Copenhagen, Denmark, 2EY Parthenon P/S, Frederiksberg, Denmark, 3Department of Child- and Adolescent Medicine, Copenhagen University Hospital, Copenhagen, Denmark.
OBJECTIVES: To evaluate real-world patient characteristics, healthcare resource utilization (HCRU), and costs among children (0-17 years) in Denmark with Spinal Muscular Atrophy (SMA) receiving disease-modifying treatment (DMT) with focus on oral risdiplam.
METHODS: This retrospective analysis (2018-2024) with data from the Danish National Patient Register and the Hospital Medicine Register evaluated children with SMA receiving a DMT, focusing on risdiplam and stratified into DMT-naïve and switch cohorts (prior DMT exposure (nusinersen)). Annualized HCRU (outpatient & inpatient visits) and healthcare costs (weighted unit costs of 2024 Danish Diagnose Related Group system) were compared pre-index (≤24 months before first risdiplam prescription) and post-index (≥30 days post-initiation of risdiplam) using descriptive statistics and non-parametric statistical testing (Wilcoxon Signed-Rank and Mann-Whitney U tests).
RESULTS: Of 57 receiving DMTs (46% females), 32 were treated with risdiplam (21 DMT-naïve; 11 switch). Diagnosis average age was 2.1 years, with DMT initiation at 9.6 years for risdiplam-treated. Comorbidities were prevalent, including respiratory problems (70%) and orthopedic complications (46%). Baseline HCRU was significantly higher in the switch cohort. Following transition to risdiplam, children in the switch cohort exhibited significant reductions in annualized outpatient visits (36.4 to 26.0; P=0.002) and inpatient admissions (3.5 to 2.5; P=0.006). Conversely, DMT-naïve children demonstrated increased outpatient visits (15.0 to 19.1; P=0.002), reflecting first-ever DMT monitoring requirements, alongside a significant reduction in inpatient admissions (P=0.014). Switching to risdiplam was associated with mean annual healthcare cost savings (€7,976 per child), while costs for DMT-naïve remained stable. A limitation is the age difference between the groups at treatment initiation.
CONCLUSIONS: Real-world data from Danish national registries demonstrate that risdiplam therapy is associated with favorable trends in HCRU. Most pronounced cost savings and reductions in hospital visits were observed in the switch cohort, while these remained stable for DMT-naïve children. Furthermore, decreased inpatient admissions were seen in both cohorts.
METHODS: This retrospective analysis (2018-2024) with data from the Danish National Patient Register and the Hospital Medicine Register evaluated children with SMA receiving a DMT, focusing on risdiplam and stratified into DMT-naïve and switch cohorts (prior DMT exposure (nusinersen)). Annualized HCRU (outpatient & inpatient visits) and healthcare costs (weighted unit costs of 2024 Danish Diagnose Related Group system) were compared pre-index (≤24 months before first risdiplam prescription) and post-index (≥30 days post-initiation of risdiplam) using descriptive statistics and non-parametric statistical testing (Wilcoxon Signed-Rank and Mann-Whitney U tests).
RESULTS: Of 57 receiving DMTs (46% females), 32 were treated with risdiplam (21 DMT-naïve; 11 switch). Diagnosis average age was 2.1 years, with DMT initiation at 9.6 years for risdiplam-treated. Comorbidities were prevalent, including respiratory problems (70%) and orthopedic complications (46%). Baseline HCRU was significantly higher in the switch cohort. Following transition to risdiplam, children in the switch cohort exhibited significant reductions in annualized outpatient visits (36.4 to 26.0; P=0.002) and inpatient admissions (3.5 to 2.5; P=0.006). Conversely, DMT-naïve children demonstrated increased outpatient visits (15.0 to 19.1; P=0.002), reflecting first-ever DMT monitoring requirements, alongside a significant reduction in inpatient admissions (P=0.014). Switching to risdiplam was associated with mean annual healthcare cost savings (€7,976 per child), while costs for DMT-naïve remained stable. A limitation is the age difference between the groups at treatment initiation.
CONCLUSIONS: Real-world data from Danish national registries demonstrate that risdiplam therapy is associated with favorable trends in HCRU. Most pronounced cost savings and reductions in hospital visits were observed in the switch cohort, while these remained stable for DMT-naïve children. Furthermore, decreased inpatient admissions were seen in both cohorts.
Conference/Value in Health Info
2026-11, ISPOR Europe 2026, Vienna, Austria
Value in Health, Volume 29, Issue 12S
Code
EE511
Topic
Economic Evaluation
Topic Subcategory
Cost/Cost of Illness/Resource Use Studies
Disease
Neurological Disorders