The EMA has launched a new pilot programme for expert panels to support the development and assessment of orphan medical devices in the European Union (EU).
This pilot programme is part of EMA’s regulatory support for the expert panels on medical devices, following the introduction of new legislation in the EU.
Since 1 March 2022, the Agency 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.
In June 2024, the European Commission published new guidance on the clinical evaluation of orphan medical devices issued by the Medical Device Coordination Group. 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 aims to guide manufacturers and notified bodies when applying the clinical evidence requirements.
What is an orphan device?
Orphan devices are medical devices which are intended to be used for diseases or conditions affecting only a small number of individuals each year (not more than 12,000 individuals in the EU per year). 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.
What does the pilot programme offer?
The pilot programme offers free advice from the medical device expert panels to selected manufacturers and notified bodies on the orphan device status and the data needed for their clinical evaluation.
The orphan device pilot will run in parallel to the scientific advice pilot to manufacturers which already prioritised advice to manufacturers on the clinical development strategy and clinical investigations of devices addressing unmet needs.
Until what date is the pilot programme expected to run?
While the pilot programme is currently scheduled to run until the end of 2025, the aim is to establish a long-term process for orphan device support.
Will the pilot programme prioritise any orphan medical devices?
As part of the pilot programme, the EMA will prioritise certain types of orphan medical devices, such as the following:
- devices for treating a medical condition that is life-threatening or that could cause permanent impairment of a body function,
- devices intended for children,
- novel devices with potential major clinical benefit.
About the expert panels in the pilot programme
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 time points in the ongoing conformity assessment of the device.
Source: EMA