Today more than ever, researchers are focused on providing care for the approximately 7,000 rare diseases that have been overlooked in the past due to the challenges of conducting clinical trials in small populations with limited commercialization potential. The orphan drug arena focuses on identifying and studying potential treatments to serve the significant unmet needs of patients living with rare diseases, and the current landscape offers a variety of incentives, government support, and regulatory agency assistance to encourage orphan drug development.
In this white paper, we will discuss key strategic considerations for both the U.S. and EU that may be employed to accelerate your orphan drug and rare disease development program. We will focus on the regulatory aspects of bringing therapies for rare diseases to market, with specific emphasis on the similarities and differences between the regulatory pathways in the U.S. and EU. We will also explore the operational nuances of orphan drug development that can help sponsors position their promising compounds for clinical and commercial success.