Successful rare disease product development requires an efficient regulatory approval strategy, designing and conducting high-quality trials that generate reliable, GCP-compliant data, and incorporating creative enrollment solutions for these hard-to-reach patient populations.

Obtaining Orphan Drug designation (ODD) from the U.S. FDA is a critical step when developing rare disease therapies. This designation strengthens the business case and investment incentive for sponsors and can help accelerate movement from preclinical to clinical testing.

Benefits of ODD include:

• Seven years of marketing exclusivity
• Tax credits
• PDUFA fee exemption
• Regulatory support from the Office of Orphan Product Development (OOPD)
• Interest from the investment community
• Access to the OOPD grant programs

Amarex's regulatory team has helped several clients obtain ODD and Breakthrough Therapy designations from the U.S. FDA. Our clinical operations department has experience conducting both adult and pediatric rare disease clinical trials. We have worked on 20-plus clinical trial projects, helping our clients with marketing approval strategy creation, IND applications and submissions, and providing full clinical trial services. Our rare disease therapeutic and specialty experience includes CNS disorders, cell and gene therapies, hematology, HIV, immunology, oncology, pain management, and wound healing.