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EC MDCG: Guidance on Clinical Evaluation of Orphan Medical Devices

The European Commission Medical Device Coordination Group (MDCG) has issued a new guidance "Clinical Evaluation of Orphan Medical Devices", which focuses on the clinical evaluation of orphan medical devices. This guidance aims to provide clear instructions and recommendations for manufacturers and stakeholders involved in the development and evaluation of medical devices intended for rare diseases, ensuring their safety, efficacy, and regulatory compliance within the European Union (EU). These devices are vital in meeting unmet medical needs and are intended for conditions affecting a small number of individuals annually.


Orphan Medical Devices: Medical devices the device is specifically intended to benefit patients in the treatment, diagnosis, or prevention of a disease or condition that presents in not more than 12,000 individuals in the European Union per year; and at least one of the following criteria are met:

  • there is an insufficiency of available alternative options for the treatment, diagnosis, or prevention of this disease/condition, or

  • 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, diagnosis, or prevention of this disease/condition, taking into account both device and patient population-specific factors.


The guidance aims to support manufacturers in the clinical evaluation of orphan medical devices by providing a framework for collecting and analyzing clinical data, addressing the specific challenges associated with rare diseases, and ensuring compliance with the Medical Devices Regulation (MDR).


A manufacturer who claims that his device is an OD should provide information that supports the OD status. The information justifying the OD status should be based on a scientific rationale addressing at least epidemiological and device-related considerations. The information justifying the OD status should be based on a scientific rationale addressing at least epidemiological and device-related considerations.


The requirements for the clinical evaluation of medical devices laid down in Article 61 and Annex XIV of the MDR also apply to orphan devices. These include the following steps:

  • establish and update a clinical evaluation plan;

  • identify relevant clinical data and any limitations in clinical data;

  • appraise all relevant clinical data; analyze all relevant clinical data;

  • generate new or additional clinical data needed to address outstanding issues;

  • document this evaluation in a clinical evaluation report; and

  • update the clinical evaluation through PMCF activities.


By addressing the unique challenges associated with rare diseases, this guidance supports manufacturers in developing robust clinical evaluation strategies and maintaining high standards of patient care.

For further details, please refer to the full guidance document here.


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