European HTA integrated evidence generation planning for a paediatric orphan therapy ​

• The client had acquired a new cell therapy for the treatment of an orphan paediatric ophthalmology indication and required alignment regarding requirements for the phase III clinical development programme​
• This needed to reflect clinical feasibility (considering the challenges with conducting a controlled trial in the target population and the realistic duration of follow-up) and the predicted evidence requirements for HTA-relevant clinical and economic value ​

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• Determine key requirements for the phase III pivotal trial design for the new cell therapy for the treatment of an orphan paediatric ophthalmology indication (to support optimal European HTA outcomes and pricing potential)​
• Define required justification to European HTA authorities regarding the rationale for the clinical development strategy (considering the challenges with clinical trial design in the target population)​
• Define additional evidence generation requirements outside of the phase III study to address parameters for HTA value demonstration (including ITCs)​

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• We completed a comprehensive review of the current and evolving therapeutic landscape for the target patient population (for advisor stimulus and to inform internal education within the company)​
• We facilitated an advisory meeting series including clinical KOLs and HTA/payer stakeholders across priority European markets​
• Findings were synthesised to inform critical requirements for the phase III trial design and ITCs to meet European HTA requirements for incremental value demonstration ​

• The client received a comprehensive review of the current and evolving therapeutic landscape for target patients (and predicted positioning for the new therapy)​
• Detailed recommendations were provided regarding key requirements for the phase III pivotal trial (and required justification for the clinical development strategy) and additional evidence synthesis to inform HTA ​
• Findings were disseminated to the cross-functional team to ensure awareness regarding the expectations for ‘clinically relevant value’ and implications for evidence generation/synthesis planning ​