Driving the Rare Disease Innovation Pipeline to Bring New Products to Market

The NIH estimates indicate that about 1 in 10 people in the United States have a rare disease, and recent studies point to an incidence of 4% to 6% worldwide. Although many rare diseases are associated with potentially correctable monogenetic defects, only a small percentage have products in development, and an even smaller percentage have approved therapies, leaving a significant unmet need for drug development in this area. However, getting new products to market can be a challenge because of the necessity for companies to raise adequate funding to support R&D in the face of inherent risks and economic realities of targeting small patient populations. This session will bring together perspectives from R&D, legal and business sectors to examine these challenges, and to discuss ways in which regulatory and orphan drug exclusivity together with patent protection and other IP create business value that helps drive the rare disease innovation pipeline to bring new products to market.