Assessing Risdiplam Cost-Effectiveness for Spinal Muscular Atrophy Types I, II, and III in Chile
Author(s)
David-Steven Góngora, MSc1, Camilo Tamayo, MSc2, Ida Caterina García-Appendini, PhD1.
1RWI IQVIA Latam, Ciudad de México, Mexico, 2RWI IQVIA Latam, Bogotá, Colombia.
1RWI IQVIA Latam, Ciudad de México, Mexico, 2RWI IQVIA Latam, Bogotá, Colombia.
Presentation Documents
OBJECTIVES: This study evaluates the cost-effectiveness of risdiplam compared to nusinersen and onasemnogene abeparvovec (AVXS-101) in patients with Spinal Muscular Atrophy (SMA) types I, II, & III over a 10-year horizon from a societal perspective in Chile, incorporating both direct healthcare costs and indirect costs related to productivity loss.
METHODS: Using an adapted Markov model, transitions between health states were assessed, accounting for disease progression and associated costs. Data on effectiveness and safety were derived from SUNFISH and FIREFISH studies, with indirect comparisons employed. Clinical outcomes were measured in terms of quality-adjusted life years (QALYs), while economic outcomes were assessed through total costs per technology, yielding incremental cost-effectiveness ratios (ICERs). Costs included acquisition, administration, medical equipment, hospitalization, follow-up, and productivity losses. A discount rate of 3% was used for both cost and clinical outcomes, and a determinist (one-way) and probabilistic sensibility analysis were performed to check the robustness of the results.
RESULTS: Results indicate risdiplam dominance over comparators across SMA types. For SMA type I, risdiplam showed a ΔQALYs of 1.40 against nusinersen and 0.04 compared with AVXS-101 and negative incremental costs, rendering it dominant. For SMA types II & III, risdiplam remained dominant with a ΔQALYs of 0.12 and reduced costs compared with nusinersen. The deterministic and probabilistic sensibility analysis show consistent results, maintaining risdiplam as a dominant alternative over its comparators.
CONCLUSIONS: In conclusion, risdiplam emerges as the preferred option for SMA patients, demonstrating superior effectiveness and cost advantages over alternative treatments. Sensitivity analyses confirm the robustness of the base case findings, reinforcing risdiplam's dominance. From a cost-effectiveness perspective, risdiplam offers the most convenient alternative for the Chilean public health system in all SMA types.
METHODS: Using an adapted Markov model, transitions between health states were assessed, accounting for disease progression and associated costs. Data on effectiveness and safety were derived from SUNFISH and FIREFISH studies, with indirect comparisons employed. Clinical outcomes were measured in terms of quality-adjusted life years (QALYs), while economic outcomes were assessed through total costs per technology, yielding incremental cost-effectiveness ratios (ICERs). Costs included acquisition, administration, medical equipment, hospitalization, follow-up, and productivity losses. A discount rate of 3% was used for both cost and clinical outcomes, and a determinist (one-way) and probabilistic sensibility analysis were performed to check the robustness of the results.
RESULTS: Results indicate risdiplam dominance over comparators across SMA types. For SMA type I, risdiplam showed a ΔQALYs of 1.40 against nusinersen and 0.04 compared with AVXS-101 and negative incremental costs, rendering it dominant. For SMA types II & III, risdiplam remained dominant with a ΔQALYs of 0.12 and reduced costs compared with nusinersen. The deterministic and probabilistic sensibility analysis show consistent results, maintaining risdiplam as a dominant alternative over its comparators.
CONCLUSIONS: In conclusion, risdiplam emerges as the preferred option for SMA patients, demonstrating superior effectiveness and cost advantages over alternative treatments. Sensitivity analyses confirm the robustness of the base case findings, reinforcing risdiplam's dominance. From a cost-effectiveness perspective, risdiplam offers the most convenient alternative for the Chilean public health system in all SMA types.
Conference/Value in Health Info
2025-05, ISPOR 2025, Montréal, Quebec, CA
Value in Health, Volume 28, Issue S1
Code
EE235
Topic
Economic Evaluation
Disease
SDC: Musculoskeletal Disorders (Arthritis, Bone Disorders, Osteoporosis, Other Musculoskeletal), SDC: Neurological Disorders, SDC: Rare & Orphan Diseases