The European Commission has released new guidance, namely “Clinical evaluation of orphan medical devices”, aimed at facilitating the clinical evaluation of orphan medical devices. Orphan devices are designed to treat rare diseases affecting no more than 12,000 individuals annually within the EU. These devices often address unmet medical needs where few diagnostic or therapeutic options exist.
Key highlights of this guidance are:
• Establish criteria for orphan status: to qualify as an orphan device, the product must target a condition affecting a small patient population (no more than 12,000 individuals in the EU per year) and offer significant clinical benefits over existing treatments.
• Clinical data requirements: while a limited amount of pre-market clinical data is acceptable for these devices, manufacturers must justify any limited amount of submitted data.
• Guidance for manufacturers: the guidance provides detailed instructions for manufacturers on justifying orphan status, using non-clinical and off-label use data, and addressing the challenges of conducting clinical investigations with small patient populations.
• Post-market surveillance: post-market clinical follow-up activities plays a pivotal role in ensuring ongoing assessment of the device’s safety and performance.
For more detailed information, you can access the full guidance here.