On 20 November 2024, the Expert Panels published templates to assist manufacturers and notified bodies in applying for advice under the EMA's pilot programme for orphan medical devices.

Templates for advice on Orphan Medical Devices

The templates from Expert Panels are designed to streamline requests for advice regarding orphan device status and clinical data requirements:

  •  Request for advice on the orphan device status pursuant to Article 61(2) of Regulation (EU) 2017/745 and MDCG 2024-10, first published: 20 November 2024
  •  Request for advice on the clinical development strategy or clinical data required for clinical evaluation pursuant to Article 61(2) or Article 106(11) of Regulation (EU) 2017/745 and MDCG 2024-10., first published: 20 November 2024

These documents serve as critical tools to guide applicants in meeting regulatory requirements under MDR.

Overview of the Pilot Programme

The European Medicines Agency (EMA) has introduced a pilot programme to support the development and market access of orphan medical devices under the Medical Device Regulation (EU) 2017/745 (MDR). This initiative provides manufacturers and notified bodies with free scientific advice on obtaining orphan status and conducting clinical evaluations for high-risk orphan medical devices.

The pilot programme aims to facilitate the transition of legacy devices to MDR compliance and help new orphan medical devices enter the EU market. Running until the end of 2025, the programme will also set a precedent for a long-term process for orphan device support.

Pilot programme as a support for manufacturers and notified bodies

The pilot programme introduced by the EMA offers significant support for manufacturers and notified bodies involved in the development of orphan medical devices. For manufacturers, the programme assists in determining potential orphan device status and provides guidance on clinical development strategies and clinical investigation proposals. For notified bodies, the programme offers help with assessing orphan status during conformity assessments and evaluating clinical data requirements during device development. Both manufacturers and notified bodies will benefit from expert advice tailored to the specific development stage of their device.

A key feature of the programme is the advice provided by expert panels. These panels review proposals and provide detailed guidance. They evaluate whether a device qualifies as an orphan device under MDR, identify required clinical data for pre-market evaluation, justify limited clinical data, and propose strategies to address clinical uncertainties through post-market clinical follow-up activities.

The pilot programme prioritizes certain categories of orphan medical devices. Priority will be given to devices targeting life-threatening conditions or those causing permanent impairment, devices intended for pediatric use, and novel devices with significant potential clinical benefit.

Applications for the programme

Applications for the programme submitted in October 2024 will be reviewed in November 2024, with four applications selected for initial testing. The EMA has created a streamlined application process, allowing manufacturers and notified bodies to submit letters of interest through a dedicated portal. These applications should clearly outline issues related to orphan status, the clinical data required, and plans for post-market clinical follow-up to address any limitations in pre-market data.

To assist applicants, templates are available to ensure clarity and consistency in meeting MDR requirements. These documents provide structured guidance for presenting clinical evaluation data and justifications for orphan device status.

Additionally, the Medical Devices Coordination Group (MDCG) published guidance in June 2024 to further aid applicants. This guidance explains the criteria for defining orphan medical devices and outlines standards for clinical evaluation under MDR Article 61(2). Manufacturers and notified bodies can use this guidance as a benchmark to ensure compliance and meet regulatory expectations effectively.

The EMA’s pilot programme provides a critical opportunity for manufacturers and notified bodies to bring innovative orphan devices to the market under MDR compliance. By offering targeted advice on orphan status and clinical data requirements, the initiative ensures these devices meet rigorous regulatory standards while addressing unmet medical needs.

For more details and to apply, visit the EMA’s portal:

https://www.ema.europa.eu/en/human-regulatory-overview/medical-devices