Rapid market opportunity assessment for an ATMP
Analysis of the clinical and commercial opportunity for a new gene therapy for a rare ophthalmology condition

This project utilised the API platform
Scope:
Global
Therapy Area:
Ophthalmology
Client:
Global pharmaceutical company
The Challenge
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
Project objectives
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
Approach
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 API project team, to inform a final report on the clinical/commercial opportunity and critical success factors for the asset
VALUE DELIVERED
The client was provided with a comprehensive assessment of the clinical and commercial opportunity for the new gene therapy for the treatment of a rare inherited ophthalmic disease, and critical success factors across key European markets to achieve commercial objectives

Insights directly addressed practical considerations for treatment delivery in hospitals, and how to achieve sustainable reimbursement for payers

The full research programme and analysis were completed within two weeks, aligning with the timelines for the business development team