Presentation (CTI)

OBJECTIVES: Sonpiretigene isteparvovec (hereafter sonpiretigene) is an adeno-associated virus serotype 2 intravitreally-administered optogenetic therapy for retinitis pigmentosa (RP) patients with severe vision loss. In 2025, the Institute for Clinical and Economic Review (ICER) estimated the average annual budget impact of a one-time, one-eye injection at $464,104 per patient. This analysis expands on ICER’s budget-impact analysis by adopting a US payer perspective, incorporating statutory rebates, and assessing the budget impact of treating both eyes and varying prevalence estimates.
METHODS: A budget impact model compared healthcare costs over a one year time horizon. Eligible population estimates (prevalence: 0.025%; proportion with severe vision loss: 12%), market shares assumptions (20% treatment initiation in the first year), and drug costs from ICER were used. Medicaid was used as an example US payer, with a 23.1% statutory Medicaid rebate and yielding 2,483 eligible patients. Outcomes included average per-member-per-month (PMPM) impact, annual impact per patient, and absolute annual impact. Sensitivity analyses assessed the impact of the proportion of unilateral and bilateral treated patients, varied prevalence estimates (0.020%-0.033%) and the proportion with severe vision loss (10%-25%).
RESULTS: In the base case for Medicaid population, the PMPM, annual per patient, and absolute annual costs were $0.18, $356,736, $177,142,763, respectively. Bilateral treatment (when applied to 100% of patients) increased PMPM costs to $0.36. At maximum prevalence and proportion with severe vision loss estimates PMPM costs increased to $0.24 and $0.37, respectively.
CONCLUSIONS: For payers, PMPM costs for treating RP patients with severe vision loss using sonpiretigene are reasonable, even under high prevalence, bilateral treatment, and proportion with severe vision loss assumptions.