European Medicines Agency Unveil Orphan Medical Device Pilot Program

The European Medicines Agency (EMA) has launched a new pilot program to support manufacturers and notified bodies on the development and assessment of orphan medical devices in the European Union (EU).

“Early advice to manufacturers, particularly to small and medium-sized enterprises, is a key tool to foster innovation and accessibility to safer and effective devices that address patients’ needs,” EMA wrote in the press release.

Orphan devices are characterized as medical devices intended to be used for diseases or conditions affecting a small number of patients each year — 12,000 or less in the EU per year. These devices are often used to treat or diagnose rare conditions which have insufficient alternative diagnostic or therapeutic options available, fulfilling a previously unmet medical need.

The pilot program will offer free advice from medical device expert panels to selected manufacturers and notified bodies on device status and the data needed for clinical evaluation. Manufacturers can consult with the expert panels throughout the different stages of device development. Notified bodies can request advice at specific moments on an ongoing conformity assessment for a device.

EMA said it will prioritize orphan devices that treat a medical condition that is life-threatening or that could cause permanent impairment of a body function, devices intended for children, and novel devices with potential major clinical benefit.

The announcement of the pilot program comes after the European Commission unveiled new guidance on the clinical evaluation of orphan medical devices issued by the Medical Device Coordination Group in June. The guidance details criteria on how to determine whether a medical device should be characterized as an orphan device under the EU Medical Devices Regulation and guides manufacturers and notified bodies when applying clinical evidence requirements, according to EMA.

Scheduled to run until the end of 2025, EMA said that after the end of the pilot program, it aims to establish a long-term process for orphan device support. The pilot will run parallel to scientific advice pilot to manufacturers which has prioritized advice to manufacturers on the clinical development strategy and clinical investigations of devices addressing unmet needs.