Concise and accurate Orphan Drug Designation Requests are critical to allow FDA to perform a thorough review and make an informed decision on the potential orphan status of a drug or biologic. This session will discuss strategic approaches to ensure the appropriate information is included in an ODD application to meet the FDA requirements focused on the areas of scientific rationale and population estimation. Some common pitfalls for these two critical sections will be discussed.
Learning Objectives:
Identify the required content of the scientific rationale and population estimate sections of an Orphan Drug Designation Request.
Describe some common pitfalls observed in Orphan Drug Designation Requests with regard to the scientific rationale and population estimate sections.
Gain further awareness of the experience of others addressing issues as a result of FDA feedback.