EMA pilot aims to advise on orphan device development

Medical device manufacturers and notified bodies in the European Union may qualify for free advice on orphan medical devices under a new pilot program launched by the European Medicines Agency (EMA). The agency will use a risk-based approach to select devices for the pilot program, and if successful, will be used as a permanent model to help bring orphan products to market.

On 2 August, EMA announced it was launching the pilot program as part of its mandate under a 2022 law where stakeholders can ask for product development advice from its expert panels. It applies to medical devices intended to treat or diagnose rare diseases and conditions that affect up to 12,000 people in the EU annually.

“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,” said EMA in a statement. “As part of the pilot program, EMA will prioritize 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.”

“Early advice to manufacturers, particularly to small and medium-sized enterprises, is a key tool to foster innovation and accessibility to safer and effective devices that address patients’ needs,” the agency added. “The orphan device pilot will run in parallel to the scientific advice pilot to manufacturers which already prioritized advice to manufacturers on the clinical development strategy and clinical investigations of devices addressing unmet needs.”

The European Commission published a guidance in June that details how it determines when a medical device should be regarded as an orphan device and how the Medical Device Coordination Group (MDCG) plans to clinically evaluate such products.

The agency emphasized that stakeholders in the pilot program will get expert advice on the status of their orphan devices, clinical development strategies, how to conduct clinical investigations for the product, and the data that needs to be collected for a clinical evaluation. Though the pilot is only meant to run until the end of 2025, the hope is that it will set the foundation for a more permanent program to allow the agency to advise manufacturers and notified bodies about orphan device development.

Stakeholders can write to EMA to request participation in the pilot program immediately. The agency also announced hosting a virtual joint meeting on 23 September with the European Commission, MDCG, and expert panels to educate stakeholders about the program. The meeting will cover topics such as definitions and criteria for orphan device status, how to address challenges with evidence generation, how to ask for scientific support from the expert panels and a demonstration of how the agency’s portal to request expert advice works.

EMA announcement

EMA pilot aims to advise on orphan device development

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