New EU plan to boost orphan medical devices

The European Medicines Agency (EMA) has launched a pilot programme to support the development and assessment of orphan medical devices in the European Union (EU). The initiative offers free advice from medical device expert panels on orphan device status and the data needed for their clinical evaluation.

This pilot is part of EMA’s regulatory support for medical devices, following the introduction of new legislation in the EU in 2022. The plan could bring new products to people with rare diseases and conditions with unmet need.

Orphan devices are medical devices which are intended to be used for diseases or conditions affecting only a small number of individuals each year (fewer 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.

The programme will initially benefit selected manufacturers and notified bodies – the national authorities responsible for assessing devices and awarding the CE mark. While the first phase of the programme is currently scheduled to run until the end of 2025, the Agency said it aims to establish a long-term process for orphan device support.

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 programme, EMA will prioritise 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.

In June 2024, 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 sets out 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 national authorities.