OBJECTIVES: Spinal muscular atrophy (SMA) is a rare genetic condition characterized by the loss of motoneurons and progressive muscle weakness.

Despite availability of 3 therapies in Poland, nusinersen since 2019, risdiplam and onasemnogene abeparvovec (OA) since 2022, there still remains high unmet need for effective treatment. This is due, among other things, to the very restricted reimbursement indication of risdiplam, available from 2 months of age and with contraindications to use nusinersen.

The aim of this study is to calculate the potential budget impact of extended reimbursement indication.

METHODS: Available National Health Fund (NHF) reimbursement data was analysed to see how many patients started therapy with risdiplam under the current, restricted indication.

The target population for the budget impact analysis covers newborn SMA patients with 1-4 SMN2 copies. Current market shares of nusinersen and OA and estimated uptake of risdiplam were estimated based on the local NHF data and clinical experts’ opinion. Two cost categories were included in the budget impact analysis: acquisition costs based on official list prices and administration costs based on the current NHF tariff. A time horizon of 2 years was adopted using the public payer perspective.

RESULTS: Since its reimbursement under restricted indication in September 2022 until December 2023, risdiplam was initiated by 304 patients with the estimate of 213 who switched and 91 with the primary contraindication to nusinersen.

Cost savings resulting from the extension of the reimbursement indication for risdiplam amount to EUR 5.0M and EUR 11.5M respectively in the 1st and 2nd year of reimbursement for a total saving of EUR 16.5M

CONCLUSIONS: Switching from nusinersen to risdiplam confirms the high unmet need for broader availability of risdiplam therapy in Poland. Extension of risdiplam’s indication to newborn patients with 1-4 SMN2 copies results in cost savings to the healthcare system.