The budget impact decreases each year as single-dose therapy precludes long-term acquisition and administration costs of treating prevalent patients. Inclusion of AVXS-101 on a formulary is estimated to offset payers' SMA care costs by -$15K by the fifth year. Additionally, installment plan for AVXS-101 payments will reduce the budget impact further.
Cost-effectiveness was dependent on assumptions made about survival, costs, and utilities; and whether the motor function milestones were sustained over lifetime. Given the relatively short-term effectiveness data available for these therapies, a registry to collect long-term data of infantile-onset SMA patients is recommended.
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