OBJECTIVES: A Managed Access Protocol (MAP) imposes eligibility criteria attached to reimbursement support for a medicine. The intention of MAPs is to provide a measure of cost certainty to the decision maker, while enabling access to innovative medicines. This study provides an overview of the MAPs for medicines for spinal muscular atrophy (SMA) managed by the Health Service Executive Medicines Management Program (MMP).

METHODS: We review the eligibility criteria applied for medicines for SMA (nusinersen, onasemnogene abeparvovec [OA], risdiplam) under the MAP approach in Ireland and provide an overview of applications received to date. Analyses were conducted in Microsoft Excel™. Data sources were the published MAPs and MMP application data.

RESULTS: All three MAPs specify that treatments can only be prescribed by approved prescribers, i.e. consultant neurologists. The diagnostic and severity criteria differ between nusinersen/risdiplam and OA, in line with the distinct reimbursement recommendations; nusinersen/risdiplam can be used in those with a clinical diagnosis of Type 1, 2 or 3 SMA aged < 18 years at initiation, while OA can be used in those with symptomatic Type 1 SMA, and pre-symptomatic SMA with up to three copies of the SMN2 gene. Genetic evidence of a diagnosis of SMA is required for all three MAPs. The requirements for the collection of outcome data are aligned across the three MAPs.

By December 2023 there were four approved prescribers under these protocols, and 69 applications had been submitted. The mean age of applicants was 7.3 years at date of application. The majority of applications were for patients with Type 1 or Type 2 SMA.

CONCLUSIONS: MAPs have been used to facilitate access to medicines for SMA in Ireland, while providing the healthcare payer with certainty that treatments will only be reimbursed in the approved cohorts aligned with those considered in the formal pricing and reimbursement process.