Critical Success Factors For Launching Products With Orphan Drug Designation

By Sid Agrawal, Dianna Cohen, and Andrew Therrien, EVERSANA

The 1983 Orphan Drug Act (ODA) was passed to financially incentivize the development of drugs for rare diseases or conditions, defined as a disease or condition that affects less than 200,000 people in the US. A recent analysis found that pharmaceutical companies that received orphan drug designation experienced increased value as measured through increases in stock price.

Despite the financial incentives for the development process, there are unique challenges to launching products with orphan indications. Conducting relevant clinical trials is a major barrier given the small patient population for many of these rare diseases or conditions. Low community awareness and lack of medical expertise can also lead to limited patient support and advocacy. This may require more personalized patient support to improve access to providers and treatments.

While receiving orphan drug designation has no impact on regulatory requirements for market authorization, there are alternate approval processes available which can ease the burden of certain regulatory elements, such as application review time or clinical trial parameters. Download the complete White Paper in PDF to explore the opportunities and strategies for orphan drug development that can maximize your potential for success.

Interested in learning more about how to advance the commercialization of rare and orphan drugs? Click here to access the solutions that are creating better patient experiences and bringing effective therapies to patients around the world.