Orphan medical devices are used to treat or diagnose rare diseases affecting fewer than 12,000 individuals annually in the EU. These devices are often essential for addressing unmet medical needs, where no or limited alternative therapies or diagnostics exist.
The European Medicines Agency (EMA) has introduced a pilot program to support the development and assessment of orphan medical devices in the EU. This initiative provides free advice from medical device expert panels to selected manufacturers and notified bodies, focusing on orphan device status and the necessary clinical data for evaluation.
Manufacturers can seek advice from expert panels at different stages of clinical strategy development, while notified bodies can request guidance during the conformity assessment process. The program will prioritize specific orphan devices, such as those designed for life-threatening conditions, devices for children, and novel devices with significant clinical potential. In June 2024, the European Commission released new guidance on the clinical evaluation of orphan medical devices. This guidance, developed by the Medical Device Coordination Group (MDCG 2024-10), helps manufacturers and notified bodies understand when a medical device qualifies as an orphan under the EU Medical Devices Regulation. It also provides guidance on specific aspects of generating clinical data and conducting clinical evaluation for this category of medical devices, as well as highlights key procedural considerations.
The pilot program is part of EMA’s broader regulatory framework, which includes support for expert panels on medical devices introduced by new EU legislation. The long-term goal is to streamline the process for orphan devices, ensuring that manufacturers can bring these crucial products to market more efficiently.
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