The client required a due diligence assessment of the European pricing, access and uptake opportunity for a new gene therapy for the treatment of a rare inherited disease leading to profound permanent vision loss for the majority of patients

The client needed to understand the clinical and commercial potential for the new gene therapy, as well as the practicalities of ensuring patient access whilst managing sustainable budget impact for European healthcare systems

Understand physician perceptions of the pivotal clinical trial design for the gene therapy, and implications for prescribing and positioning in the treatment pathway

Understand likely HTA outcomes based on the clinical data package, and implications for pricing potential

Analyse acceptable payment/financial models for European payers to support sustainable access for high cost ‘curative’ gene therapies

Explore likely payer management of the gene therapy, including the funding source/pathway and predicted restrictions on patient access

A desk research programme was completed to understand current treatment guidelines for the condition, epidemiology, and key trends in the clinical/competitive landscape

Primary research was completed with 10 KOLs in the relevant rare ophthalmology disorder (to understand the value of the product profile, and likely use) and 12 payers across the EU5 countries (to understand the HTA/pricing landscape and likely payer management)

Research findings were analysed by the Lightning Health project team, to inform a final report on the clinical/commercial opportunity and critical success factors for the asset