European HTA requirements and evidence generation/synthesis for a new gene therapy​

• A mid-size biotech company was developing a new gene therapy for a rare haematology indication, and required a detailed understanding of the European HTA requirements to support long-term value (and a likely curative effect)​
• This was needed to directly inform further evidence synthesis activities, assumptions for cost-effectiveness model development, and justification for the European pricing strategy (and preparation for a potential ICER submission in the US) ​

• Define the minimum data requirements to support reimbursed patient access for the new gene therapy across priority European markets (considering introduction of the JCA)​
• Seek HTA stakeholder alignment regarding requirements for the credible extrapolation of the long-term clinical value of the gene therapy over the lifetime horizon for the patient (assuming treatment is administered in children and has a potentially curative effect)​
• Define prioritised action planning for further evidence generation/synthesis activities ​

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• Virtual advisory meetings were organised according to European HTA archetypes (cost-effectiveness and clinical-effectiveness led decision making); each advisory meeting also included a European KOL​
• All advisors received a detailed stimulus package regarding the therapy area and proposed data package for the gene therapy​
• The meetings were moderated by the Lightning Health team, to seek areas of consensus and divergence regarding requirements for European HTA value demonstration ​

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• The client received a detailed assessment of the implications for the proposed clinical/economic evidence package for European HTA outcomes ​
• This directly informed prioritisation of further evidence generation/synthesis/expert elicitation activities to justify claims regarding the long-term curative effect of treatment across the lifetime horizon​
• This also directly informed the proposed approach for modelling a potential cure within HTA submissions​

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