European value & access/launch strategy development for a new therapy for an ultra-orphan genetic disorder ​

• The company was in phase II development for a novel therapy targeting an ultra-orphan condition with limited available treatment options and a high burden of disease​
• The commercial, HEOR and value & access teams required a detailed understanding of the opportunities and risks for pursuing European market access based on phase II data, the required clinical trial design, implications for pricing/access and likely requirements for RWE collection within an integrated European value & access strategy ​

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• Define critical success factors for the European launch for the new therapy ​
• Inform the key requirements from the phase II trial to support patient access (assuming conditional approval) and optimal pricing across priority European markets ​
• Define the likely net pricing potential (considering the likely pricing comparator, and scenarios for incremental clinical/humanistic/economic value)​
• Define the cross-functional tactical plan required for implementation of the value & access strategy/launch programme ​

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• Research with clinical/HTA stakeholders across the EU4 + UK to inform key clinical/payer value drivers for the new treatment, predicted HTA/pricing outcomes based on the proposed integrated evidence generation strategy, and requirements to optimise pricing, access and uptake​
• Internal workshops to consolidate insights into a clear asset plan for the new therapy, including implications for market segmentation, integrated evidence planning, health economic modelling and the optimal European pricing corridor ​

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• Detailed assessment of the clinical landscape and key drivers of clinical/payer value​
• European value & access strategy development, including the cross-functional plan for implementation across global and country affiliate teams (and associated timelines/dependencies and resource requirements)​
• Integrated evidence planning covering pivotal trial, evidence synthesis/RWE and health economic modelling requirements to optimise clinical/commercial value ​

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