Phase III trial support and JCA planning for an innovative therapy for a rare neurological disorder ​

• The client was developing a clinical innovation for a rare neurological disorder. To support timely patient access globally, the client required recommendations to optimise their Phase III trial design and support global HTA evaluation and health economic evidence generation​
• With planned launches across Europe and the US, it was also critical to align internal teams on key EU JCA processes and submission requirements, as well as on the potential implications of the evolving US pricing and market access landscape (e.g. the “Most Favoured Nation” policy) for global pricing strategy​

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• Aligning cross-functional teams on EU JCA submission requirements, including timelines, stakeholders, evidence expectations and PICO implications​
• Critically assessing the proposed Phase III trial design and its implications for HTA and reimbursement in the EU4, UK and US, informed by analogue examples​
• Sharing best practices in economic modelling and value narrative development​
• Providing an overview of the US ‘MFN’ policy and potential impact for the client’s asset​

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• We conducted a targeted analogue analysis of 5 assets, reviewing how the HEOR and clinical evidence package influenced HTA/pricing decisions across markets. Insights were synthesised into a report with actionable recommendations for the Phase III trial design and HEOR evidence strategy​
• We delivered an interactive workshop for the client cross-functional team (including market access, HEOR, regulatory, commercial) to support planning for JCA implementation, health economic value requirements and consideration of wider policy dynamics (including US MFN pricing implications)​

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• Clear guidance regarding Phase III trial requirements (and wider HEOR data generation) to optimise European HTA value demonstration (and pricing potential)​
• Operational readiness to JCA implementation (including timelines/required actions for JSC engagement and meeting key milestones for the JCA submission)​
• Awareness and understanding of risks associated with the US MFN policy and priority considerations for the pricing strategy/launch sequence ​

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