EMA 设立孤儿医疗器械专家小组支持流程

EMA has established a voluntary process enabling manufacturers of orphan medical devices and notified bodies to request support from relevant expert panels.

The support applies to all classes of devices, in line the Medical Device Coordination Group's guidance on the clinical evaluation of orphan medical devices.

It is free of charge.

This followed an expert panel support pilot that ran between 2024 and 2026.

EMA ensures the secretariat of the expert panels. It coordinates the support process and acts as the main contact point for applicants.

For more information, see:

Medical devices

How it works

For applications from manufacturers, the expert panels will advise:

regarding a possible orphan status of a device;
and on the clinical development strategy and / or proposals for clinical investigation for the respective device, for devices that are granted orphan designation.

For applications from notified bodies, the expert panels will advise:

either on a possible orphan status of a device;
or on the data required for the clinical evaluation during an ongoing conformity assessment, for devices that are granted orphan designation.

Notified bodies will submit their advice request in cooperation with the orphan device manufacturer.

The request for advice depends on the stage of development of the device.

The first step is getting an opinion on the orphan device status. This enables asking for clinical advice for devices that are granted an orphan device designation. Clinical advice may include an opinion on:

data sets required for the clinical evaluation as part of the conformity assessment;
review of the justification regarding the limited clinical data;
acceptability of clinical uncertainty and limitations in the data sets;
acceptability of the proposed post-market clinical follow-up activities.

The Paediatrics and rare diseases expert panel is the main panel involved in orphan device applications. Experts can be recruited from the other thematic panels as necessary.

For more information, see:

Medical device expert panels

Guidance on orphan medical devices regular support process

EMA guidance for manufacturers / applicants is available below, including step-by-step instructions.

Guidance is also available to help determine when a medical device or an accessory for a medical device should be regarded as ‘orphan’. This guidance provides a benchmark to manufacturers and notified bodies on the clinical evaluation of orphan devices which require clinical data evidence to demonstrate conformity with the Medical Device Regulation (Regulation (EU) 2017/745). The from Medical Devices Coordination Group (MDCG) published the guidance in June 2024.

For more information, see:

How to apply

To submit your application, follow the steps described in the expandable panels below:

You can request this type of advice if you are an EU-based manufacturer or their authorised representative, or a notified body designated under the Medical Device Regulation.

To do so, create an EMA account via the link below: 

EMA Account Management

To request advice, open the 'letter of interest' form for Orphan device according to MDCG guidance 2024-10. You can access the form through the dedicated portal linked below when you have an EMA account.

Business Services - Employee Center

Note that the link above also serves to request other types of support from the expert panels. 

To correctly complete and submit the letter of interest form, select the “Advice in scope of the MDCG guidance 2024-10” option from the corresponding drop-down list.

While you can submit a letter of interest at any time, you can only submit the required final briefing document according to the timetables available below:

Briefing document templates

Briefing document templates in PDF format are available for orphan designation and clinical advice.

Applicants receive a Word version of the template that they need to fill in during their application.

For more information, see:

Contact

Former pilot programme

EMA's pilot programme to support orphan medical devices provided free advice on a possible orphan device status and on the clinical evaluation of orphan medical devices.

The pilot focused on both new orphan medical devices and legacy medical devices, which were transitioning under rules of the Medical Device Regulation (EU) 2017/745 (MDR). Concretely, it aimed to help bring new orphan medical devices onto the EU market while keeping legacy medical devices on the market.

The pilot programme was designed to run until the end of 2025, but applications were still accepted in early 2026.

More information on the pilot programme is available in the report below:

Page update history

An update log is available to show the date and summary of changes to this webpage. It does not include updates to linked documents or minor edits like typos or broken link fixes.

The tracking of updates begins in March 2026.

27 October 2026

Report added to the 'Former pilot programme' section.

1 April 2026

Page updated to reflect the availability of an established expert panel support process for manufacturers of orphan medical devices and notified bodies. This includes new guidance for applicants, detailed information on how to apply and new briefing document templates.

2 March 2026

Page first published; Content has been moved from another webpage as part of a broader content reorganisation