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. These increased clinical evidence requirements present a challenge for devices specifically intended for use in rare diseases/conditions, or in specific indications for rare cohorts of patients with an otherwise non-rare disease/condition.
By their nature, these ‘orphan devices’ are only intended for use in a small number of individuals each year. Many rare diseases have very few diagnostic or therapeutic options and the orphan device can be particularly crucial to fulfil an otherwise unmet medical need. In the absence of specific guidance for these devices, different understandings can emerge among manufacturers, notified bodies, and regulators on the clinical evidence requirements for these devices for the purposes of MDR certification.
In many cases, orphan devices are intended for use solely or predominantly in minors and paediatric populations, and/or in emergency situations. Proactively generating clinical data within an appropriate time in small patient populations is particularly challenging, as is the case for vulnerable populations in light of the ethical and regulatory requirements to appropriately protect these populations, as well as greater practical challenges of performing clinical studies in certain cohorts such as infants and children.
The increased, and at times unpredictable, financial costs associated with compliance with MDR requirements, including MDR certification, can make it prohibitive for manufacturers to place orphan devices on the EU market, as the low volumes of sales may not offset the financial costs.
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.
MDCG 2024-10 - Clinical evaluation of orphan medical devices - June 2024
MDCG 2024-10 - Clinical evaluation of orphan medical devices - June 2024
health.ec.europa.eu
