The marketplace for orphan drugs is growing, and changes in the regulatory landscape are providing favorable conditions for collaboration in the area of drug development in rare diseases. Understanding the regulatory and operational nuances of orphan drug development can help sponsors position their promising compounds for clinical and commercial success.
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.
The market opportunity in rare diseases
Approximately 7,000 distinct rare diseases have been identified to date, affecting an estimated 350 million people worldwide. Since the Orphan Drug Act was passed in the U.S. in 1983, there have been over 2,900 orphan drug designations, and more than 40 orphan drugs have exceeded $1 billion in global sales. With an estimated 250 new rare diseases identified each year, the worldwide orphan drug market is projected to grow to $127 billion in 2018 and is expected to grow at an annual rate of 11 percent per year through 2024, compared with six percent for non-orphan drugs.