Managed Access Protocols As an Innovative Approach to Enabling Access to High-Cost Orphan Medicines: The Irish Experience

OBJECTIVES: A Managed Access Protocol (MAP) imposes eligibility criteria attached to reimbursement support for a medicine, and represents a form of health technology management. MAPs are increasingly used to facilitate access to orphan medicines in Ireland. This study reviews the reimbursement criteria applied in MAPs for orphan medicines managed by the Health Service Executive Medicines Management Program (HSE-MMP).

METHODS: MAPs for orphan medicines, managed by the MMP up to December 2023, were eligible for inclusion and identified via the MMP website. Data were extracted from the publicly available MAPs, and an internal MMP application database. Key features of the identified MAPs were extracted from the MAPs, categorized, and compared across the MAPs. We provide an overview of the individual reimbursement applications received under these MAPs in 2023.

RESULTS: Ten MAPs for orphan medicines were eligible for inclusion. Fifty percent (n=5) of included medicines were reimbursed in a subpopulation of the licensed population. All MAPs included a stipulation regarding prescriber eligibility, and specified diagnostic criteria. Clinical severity criteria were specified in six MAPs, while a requirement for a trial of previous line(s) of treatment with a medicinal product was specified in two MAPs. All MAPS included a provision for the collection of outcome data.

Reimbursement applications for orphan medicines reimbursed under these MAPs in 2023 were received for 118 individuals, the majority (59.3%) with a diagnosis of transthyretin amyloidosis. Reimbursement approval was granted for 94.9% of submitted applications.

CONCLUSIONS: MAPs enable equity of access to orphan medicines for patients with rare diseases who meet the eligibility criteria. MAPs facilitate access for patients with the greatest unmet clinical need; the high approval rate indicates MAPs do not represent a barrier to access. MAPs can be used to support performance-based reimbursement and provide a measure of oversight, cost certainty and cost containment for the payer.