The European Commission is proposing to establish a dedicated expert panel for orphan paediatric devices. This panel aims to provide scientific and clinical advice for devices intended for rare diseases and children. The initiative recognizes the unique challenges in developing medical devices for small patient populations, and seeks to enhance support for their development and assessment. Orphan medical devices are i) devices intended for diseases or conditions affecting no more than 12,000 individuals in the EU per year, and ii) devices that address conditions where there are insufficient alternative treatment options. They must offer expected clinical benefits compared to available alternatives.

A Specialised Focus for Rare Diseases

The European Commission plans to create a specialized expert panel focused on medical devices for orphan paediatric devices. Adoption is slated for the second quarter of 2025. This panel will join eleven existing expert panels that provide scientific, technical, and clinical opinions. These panels support the implementation of EU medical device regulations. Currently, the initiative is in the draft stage and public feedback period runs from May 19 to June 16, 2025.

The expert panel will build upon several important developments in EU medical device regulation:

• In August 2024, the European Medicines Agency (EMA) launched a pilot program. It offers free advice from medical device expert panels to manufacturers and notified bodies on orphan device status and clinical evaluation.
• The pilot program is scheduled to run until the end of 2025. The goal is to establish a long-term process for orphan device support.
• In June 2024, the Medical Device Coordination Group issued new guidance on the clinical evaluation of orphan medical devices. It provides criteria to determine when a device qualifies as an orphan device under EU regulations.

Tackling Market Limitations

The establishment of this expert panel addresses two fundamental challenges in bringing medical products for rare diseases to market: i) the difficulty in obtaining sufficient clinical data due to small patient populations for clinical studies, and ii) limited economic incentives for developing products with small markets.

Early advice to manufacturers, particularly SMEs, can also foster innovation. It can improve access to safer, more effective devices addressing patients’ needs. The panel could help ease the transition under the new Medical Device Regulation (MDR) rules. It may facilitate the introduction of new orphan paediatric devices to the EU market while preserving access for legacy devices. If the panel provides guidance on situations where it may be justified to certify products with limitations in pre-market clinical data, then it would overcome challenges with implementing large clinical trials.

Prioritizing Vulnerable Populations

The establishment of a dedicated expert panel for orphan paediatric devices represents a significant step toward addressing the unique needs of patients with rare diseases and children. By providing specialized scientific and clinical advice, the EU aims to overcome the specific challenges associated with developing medical devices for small patient populations. Ultimately, it seeks to improve access to innovative medical technologies for these vulnerable groups. For more details on this initiative, check out the EU Commission’s dedicated page.