The MDCG has released a guidance document concerning the Clinical Evaluation of Orphan Medical Devices. As defined in the guidance document, for a device to be considered an orphan device, it must be a medical device that is specifically intended to benefit patients in the treatment, diagnosis and prevention of a disease that presents in not more than 12,000 individuals per year in the EU, as well as a device that meets one of the following criteria:
- there is insufficiency of available alternative options
- the device will offer an option that will provide an expected clinical benefit compared to available alternatives or state of the art for the treatment
The level of clinical evidence that is required to place medical devices on the market has been increased by the MDR, including an increased need for pre-market clinical investigations for certain higher risk devices to verify their safety and clinical performance. As one can imagine, this increased requirement to provide more clinical evidence can present many challenges for devices that are specifically intended to be used in rare medical conditions.
Due to the aforementioned challenges facing such devices, the MDCG has published this guidance document to provide guidance to manufacturers and notified bodies on the clinical evaluation pursuant to the MDR of medical devices and accessories for medical devices that qualify as ‘orphan devices’ (OD) and medical devices and accessories for medical devices that have an orphan indication, within the meaning of this guidance.
This document is divided into two parts, and includes guidance on the following:
PART A – Clinical evaluation considerations
- The acceptability of limitations in pre-market clinical data for orphan devices,
- Key considerations on the clinical evaluation of new and legacy orphan devices,
- Generating post-market clinical data for orphan devices, including PMS and PMCF.
PART B – Procedural considerations
- Guidance for notified bodies on the assessment of orphan devices,
- The role of expert panels in the context of orphan devices.
There are three appendices to this document, which include guidance on:
- OD-specific factors to include in the clinical evaluation report,
- Consideration on clinical investigations of orphan devices,
- Extrapolation of clinical data to orphan indications
The guidance document can be accessed using the following link
