Understanding Orphan Designations and Rare Disease Studies from a Regulatory Perspective
Rare diseases are diseases which affect a small number of people compared to the general population. There are thousands of rare diseases, with 6-7,000 discovered to date and new diseases regularly described in medical literature.
Global Regulatory Expertise
A key component is global regulatory understanding and consultation regarding the unique environment for Rare Disease studies.
Orphan medicines are those intended to treat rare diseases and represent the focus of regulatory agencies in driving state-of-the-art innovation in this area. Regulatory bodies define rare diseases as those that are life-threatening or chronically debilitating conditions.
Medpace’s Innovative Approach for Rare Disease and Orphan Drug Projects
Medpace has developed experienced project teams for these complex studies. Integrated processes regarding site relationships with access to patient registries to drive patient enrollment, broad understanding of patient advocacy issues and organizations for support, and noted physicians and pediatricians are critical to a project’s success.
- What is the Regulatory Environment for Orphan Policies in Key Global Regions?
- Orphan Drug Designation Focus Areas
- Medpace Services