Orphan Medical Devices in the European Union
Regulatory Q&As for Researchers
The OrphaDev4Kids Project offers regulatory support to stakeholders developing Orphan Medical Devices. We have compiled a series of Regulatory Q&As to help you in your medical device research and development.
In this section you can find information about the EU Regulatory Context and Definitions.
If you need any additional information don’t hesitate to reach out to [email protected]
Is there a definition of “orphan medical device” in EU regulatory guidance?
The EU legislation does not currently include a formal legal definition of “orphan medical device”.
However, a working definition is provided in the guidance issued by the Medical Device Coordination Group (MDCG 2024-10). According to this guidance, a medical device or an accessory for medical device should be regarded as “orphan medical device”, if it meets the following criteria:
- 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 EU Union per
year; and at least one of the following criteria are met:
- there is 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.
More information:
How is the “orphan population” for a medical device determined?
There is flexibility in calculating the number of individuals targeted by a medical device. Both device-specific such as mechanism of action, and patient-specific factors that make it medically plausible that the device is for use only for that specific subpopulation of patients, can be considered when defining the population.
Orphan population:
If epidemiological evidence shows that the disease or condition affects no more than 12,000 individuals per year in the EU, this can be sufficient to justify the device as targeting an orphan population.
Orphan sub-population:
If the device is intended for a subset of a broader disease or condition, manufacturers can provide evidence to identify the clinically valid patient population for which the device is intended. A clear rationale for selecting this sub-population can be sufficient to support regulatory submissions.
This approach is consistent with guidance such as MDCG 2024‑10, which emphasises that the definition of the target population should be evidence-based and proportional to the clinical context.
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How is devices classification determined?
Devices classification is governed by Annex VIII MDR (Class I, IIa, IIb, III), which establishes rules based on:
- Duration of use;
- Degree of invasiveness;
- Potential risk to patients.
Application of the classification rules shall be governed by the intended purpose of the devices.
For in vitro diagnostic medical devices, classification is governed by Annex VIII of the IVDR, which establishes four risk classes (A, B, C, and D) based primarily on the intended purpose of the test and the potential public health and individual patient risks associated with incorrect results.
The guidance on classification of medical devices (MDCG 2021-24) is relevant to medical devices and provides a general overview on the impact of the classification of medical devices on different aspects of the device compliance with the legal requirements.
For IVD devices, additional MDCG guidance documents are available to support the interpretation of IVDR classification rules (MDCG 2020-16).
More information: