Onasemnogene Abeparvovec Gene Therapy and Risdiplam for the Treatment of Spinal Muscular Atrophy in Thailand: A Cost-Utility Analysis

Figures

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  Fig. 1. Markov model representing the health state transitions for SMA patients. SMA type 1 cases received either one of onasemnogene or risdiplam, while SMA types 2–3 cases received only risdiplam, compared with the standard of care (SOC). Dashed line arrows represent the transition of health states for SMA patients who received either onasemnogene or risdiplam. Solid line arrows represent the transition of health states for SMA patients who received SOC. The description of each health state is as follows: (A) a broad range of normal development (BRND; defined as able to walk independently); (B) walking with support or equivalent to SMA type 3; (C) sitting (defined as able to sit without support, stand alone, or stand with assistance) or equivalent to SMA type 2; (D) not sitting but living ventilation-free (defined as lacking head control, voluntary grasping, voluntary kicking, or roll-over completely and not requiring permanent ventilation) or equivalent to SMA type 1; (E) permanent assisted ventilation (PAV; defined as 16 or more hours per day of breathing support) or equivalent to SMA type 1; and “Death”

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  Fig. 2. Tornado diagram of one-way sensitivity analysis on the mean incremental cost-effectiveness ratio (ICER) of onasemnogene in SMA type 1 patients from the societal perspective with a 3% per annum discount. IPD inpatient department, LB lower bound, PAV permanent assisted ventilation, QALY quality-adjusted life year, UB upper bound

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  Fig. 3. Tornado diagram of one-way sensitivity analysis on the mean incremental cost-effectiveness ratio (ICER) of risdiplam in patients with SMA types 2–3 from the societal perspective with a 3% per annum discount. LB lower bound, OPD outpatient department, PAV permanent assisted ventilation, QALY quality-adjusted life year, UB upper bound

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  Fig. 4. Cost-effectiveness acceptability curve for SMA type 1 at different WTP thresholds. QALY quality-adjusted life year, SOC standard of care, WTP willingness to pay

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  Fig. 5. Cost-effectiveness acceptability curve for SMA types 2–3 at different WTP thresholds. QALY quality-adjusted life year, SOC standard of care, WTP willingness to pay