Early asset evaluation

EARLY ASSET EVALUATION OF A NOVEL THERAPY IN AN ULTRA-RARE PAEDIATRIC DISEASE*
SCOPE: EU5+US

BACKGROUND
Due to its rarity, genetic origin, high unmet need and involvement of paediatric patients, this disease represents a high-value therapeutic area.

PROJECT OBJECTIVE
To estimate the commercial opportunity associated with the development of a hypothetical early asset for the management of the disease.

APPROACH

• Analogue orphan product research (analogues indicated in a genetic condition), through EMA and FDA databases.
• Analysis of epidemiology, evidence provided at time of market approval, annual cost achieved, market access status in US + EU5, for each of the analogue products.
• Conclusions on an achievable annual cost for a therapy indicated in this disease, based on analogue experience.
• Definition of an EU price corridor, based on European referencing experience.
• Definition of value messages to support achievement of optimal pricing.

OUTPUT
The analysis showed high willingness-to-pay of US and EU payers for therapies indicated in similar conditions with relevant unmet need. The price potential for the new asset was defined for EU5 and US in the hypothesis a base case clinical value could be demonstrated.

(*) The specific disease is not mentioned due to confidentiality reasons.