Presentation (CTI)

OBJECTIVES: The Most Favoured Nation (MFN) policy aims to anchor US prices for medicines to a basket of high-income OECD reference markets, pressuring manufacturers to withdraw/delay/not consider public reimbursement in these reference markets. Much discussion has focussed on mitigating strategies (such as alternative pathways to public reimbursement) in European markets. However, for the mandatory GLOBE and GUARD models (for Medicare Part B and D, respectively) five of the nineteen reference markets sit outside Europe: Australia, Canada, Israel, Japan and South Korea. This research examines alternative access pathways in these five markets, focussing on Ultra-rare disease therapies (URTs), which often represent some of the highest priced therapies.
METHODS: Alternative access pathways for URTs were identified across these five markets in May 2026. Pathways were grouped into four categories: Named Patient Programmes (NPP), Early Access Programmes (EAP), Private Insurance, and 'Other' (including dedicated funding schemes, expanded-access clinical trials, and provincial/regional exceptional access). The presence and likelihood of reimbursement through each pathway were assessed via desk research and validated through payer and clinical expert interviews.
RESULTS: All four pathway categories were observed across the 5 markets: NPPs was present in 5/5 markets, though these provide access on an individual patient basis. Private Insurance covering high-cost drugs was available in 3/5 markets (Canada, Israel, South Korea). 'Other' pathways were also present in 3/5 markets: Australia’s Life Saving Drugs Programme, Canada’s Provincial Exceptional-Access Programme, and Japan’s Expanded-Access Clinical Trials. EAPs were the least common (2/5 markets: Australia, Japan), reflecting limited formal pre-reimbursement frameworks.
CONCLUSIONS: Public reimbursement of high-cost URTs in MFN reference markets may carry significant commercial risk. Alternative access pathways offer a way to sustain patient access and unlock commercial opportunity without anchoring a low reference price. However, availability and scope of these pathways vary by market necessitating country-specific engagement strategies.