PRO12 Early Pricing and Access Forecasting for Orphan Assets - Leveraging a Value-Driven Approach to Price Forecasting

Abstract

Orphan products could contribute to 45% of new asset launches by 2023. With recent advancement of cell and gene therapies and challenges within an increasingly challenging payer environment, ensuring a return on investment within small patient populations is increasingly challenging. Expanding on from our previously reported oncology multiple criteria decision analysis (MCDA) framework in previous ISPOR events, we have expanded the orphan MCDA based on a variety of different value attributes that drive decision-making within orphan indications to arrive at a value framework.