Developing products for rare diseases and orphan products presents unique challenges. Rho has extensive experience addressing these challenges to set your program up for success. Here are some resources we have developed to help you:
Personalized Medicine versus Orphan Product Development
The development of therapeutics for personalized medicine leverages many of the advantages of the legislation conferred upon orphan products. New products must be positioned intelligently to take advantage of this legislation. Join David Shoemaker, Ph.D. as he discusses the benefits of orphan product designation in the US, EU, and strategies for obtaining these designations for novel products.
Dr. Shoemaker has 25 years of experience in pharmaceutical development and has led the regulatory strategy for programs in many therapeutic areas. He has experience with all types of regulatory submissions and has moderated dozens of FDA meetings.
Key Tips for Developing Orphan Products
Dr. Shoemaker offers six essential tips for developing orphan products based on his extensive experience working with products intended to treat rare and orphan diseases.
White Paper: Accelerating US Regulatory Approval for Drugs and Biologics that Treat Serious Diseases
FDA’s Fast Track, Accelerated Approval, and Priority Review designations can reduce product approval timelines if pursued and implemented appropriately. Taking the time to understand the requirements and potential benefits of each acceleration mechanism will help you make informed decisions about what’s best for your product development program.