Orphan Devices - Finding Rarity: A Case Study in Achieving Orphan Device Designation in the EU

Description: Generation and demonstration of clinical evidence may be the most agonizing task of device submissions, especially under the EU MDR. Developing the regulatory strategy and clinical development strategy for a device is complex. For example, what actually is sufficient? Add to that the complexity of a device that merges multiple characteristics: active, implantable, and drug-device combination. Not complex enough? Ok, it is also indicated for a rare brain disease too.

Such a regulatory strategy requires creativity and ingenuity to find and take advantage of the flexibility within the EU MDR, like orphan device status.

This case study addresses the recently implemented pilot program concerning orphan devices, launched in July 2024.
This session focuses specifically on the benefits of the EU orphan device pathway as well as challenges in achieving this designation. We will discuss the clinical and regulatory requirements in successfully meeting European Medicines Agency Expert Panel expectations.

This session will address the designation process during pilot phase:
- defining and discriminating appropriate intended purpose, medical indications and intended population;.
- establishing and generating clinical evidence, especially when no similar devices exist;
- applying to the Expert Panel: lessons learned;
- understanding MDCG 2024-10 and how useful it was;
- planning for the future and discussing the next steps and opportunities for the devices until it is launched on the market;
- addressing process differences that apply to drugs but also to the breakthrough device program in the US;
- and eventually opening of the discussion on anticipated requirements and expected regulatory evolutions.