New European Medicines Agency Program Aims to Support Orphan Device Development

The European Medicines Agency has launched a pilot expert panel program to support orphan medical device development and assessment in the European Union (EU).

The pilot program offers free advice from medical device expert panels to selected manufacturers and notified bodies on the orphan device status and the data needed for their clinical evaluation. The pilot program is currently scheduled to run until the end of 2025, but authorities hope to establish a long-term orphan device support process.
 
Orphan devices are medical devices used to treat diseases or conditions that affect a small number of individuals every year (not more than 12,000 individuals in the EU annually). Often they are used to treat or diagnose rare diseases or conditions for which no or insufficient alternative diagnostic or therapeutic options exist, thereby fulfilling an unmet medical need.
 
Manufacturers can consult the expert panels at different stages of the development of the clinical strategy for their device, while notified bodies can request advice at specific moments of the ongoing conformity assessment of the device. As part of the pilot program, EMA will prioritize certain types of orphan medical devices, such as devices for treating 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.
 
This past June, the European Commission announced new guidance on the clinical evaluation of orphan medical devices issued by the Medical Device Coordination Group, which is composed of representatives of all EU Member States. This guidance provides the criteria to determine when a medical device should be regarded as an orphan device under the EU Medical Devices Regulation and attempts to guide manufacturers and notified bodies when applying the clinical evidence requirements.
 
This pilot program is part of EMA’s regulatory support for the expert panels on medical devices, following the introduction of new legislation in the EU. Since March 1, 2022, the sgency supports the medical device expert panels that provide opinions and views to notified bodies on the scientific assessment of clinical and performance evaluations of certain high-risk medical devices and in vitro diagnostic medical devices.
 
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. The orphan device pilot will run in parallel to the scientific advice pilot to manufacturers that already prioritizes advice to manufacturers on the clinical development strategy and clinical investigations of devices addressing unmet needs.