European health economic value demonstration strategy for a new therapy for a haematological disease ​

• The client was completing integrated evidence planning for the European launch of a new therapy for a rare haematological disorder​
• The company was planning for potential EMA conditional approval based on phase II data, and the associated implications for health economic model development in the context of high levels of uncertainty regarding the long-term clinical/economic value of the treatment ​

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• Define the structural requirements for the cost-effectiveness model for the new therapy (including required health states considering potential diverse disease trajectories based on relevant subgroups)​
• Highlight key areas of uncertainty for cost-effectiveness modelling and required assumptions/pragmatic mitigation to minimise the risks for European HTA authorities ​
• Inform the programme of evidence generation/ synthesis and further validation required within the European value & access strategy development ​

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• We reviewed the proposed health economic modelling approach and draft model structure in partnership with the HEOR team​
• An advisory meeting was completed including senior academic health economists and recent former HTA stakeholders across relevant markets (e.g., NICE) to inform the optimal approach for health economic value demonstration​
• Insights were consolidated into detailed recommendations and cross-functional action points to ensure readiness in time for European launch ​

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• The company received detailed guidance on the recommended approach for cost-effectiveness modelling in the context of high levels of uncertainty​
• This included guidance on the required health economic model structure, requirements for credible extrapolation of likely long terms clinical/QoL/economic outcomes, and implications for the statistical analysis plan ​
• Recommendations and required activities were integrated within the integrated evidence plan and European launch programme preparation ​

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