How to Successfully Navigate Orphan Drug Designation

Summary

An orphan designation may add value to a product by potentially increasing stock value, increasing the company visibility, and initiating early interactions with Health Authorities. However, seeking an orphan designation too early has its disadvantages, including public disclosure of regulatory strategy, product positioning, and risking a premature application with a following rejection due to insufficient data. As a result, when to submit an ODD is subject to particular consideration. This paper explores how to optimize this process.