Summary
This guide addresses the regulatory and procedural aspects of marketing orphan medical devices under the EU Medical Device Regulation (MDR). It defines orphan devices as those treating conditions affecting fewer than 12,000 individuals annually in the EU and outlines the typical populations served, including rare subpopulations like pediatrics. The document discusses the range of pre-market non-clinical data that can be utilized, which includes laboratory studies, computer modeling, and cadaveric studies. It emphasizes the importance of identifying data limitations and addressing them in post-market clinical follow-up (PMCF) activities. The guide also covers the possibility of data extrapolation based on scientific validity and the relevance of similarities between populations. Additionally, it highlights the benefits of structured dialogues with notified bodies and optional consultations with the EMA expert panel to confirm orphan status and assess clinical data. A checklist is provided to assist manufacturers in determining the adequacy of their information for conformity assessment applications.
