Today, 25 June 2024, the MDCG (Medical Device Coordination Group) published a guidance on the clinical evaluation of orphan medical devices (MDCG 2024-10). An orphan device is a medical device “intended for use in rare diseases/conditions, or in specific indications for rare cohorts of patients with an otherwise non-rare disease/condition”, as defined in the Guidance.
As orphan devices are designed for rare diseases or conditions and the population sample is small, collecting clinical data can be challenging.
Clinical evaluation data for orphan devices
A manufacturer who claims that their device is an orphan device should provide information that supports this status. Specifically, the manufacturer should provide a description of the specific disease or condition this device is use for as well as a description of the device, its intended purpose, and a scientific rationale for why the proposed intended use is considered necessary or important in the context of the orphan population’s management.
The requirements for clinical evaluation of medical devices laid down in Article 61 and Annex XIV of the Medical Devices Regulation also apply to orphan devices. This includes, among other things, establishing a clinical evaluation plan and identifying, appraising, and, analysing clinical data.
Nonetheless, the guidance clarifies that orphan devices may be granted market access with acceptable limitations in the pre-market clinical data amount and quality. In general, a limited level of pre-market clinical data is acceptable, if:
- All available relevant non-clinical and clinical data have been evaluated
- Existing non-clinical and limited clinical data is sufficient to demonstrate compliance with GSPRs, an acceptable benefit-risk ratio, and an expected clinical benefit
- It is not feasible or proportionate to generate further clinical data within an acceptable time frame
- The manufacturer has an adequate PMCF plan
- Device’s users will be informed adequately.
PMCF to address data limitations
If such pre-market clinical data limitations are identified and deemed acceptable, it is important that the Post-Market Clinical Follow Up (PMCF) documentation addresses these limitations through well-defined and structured PMCF activities.
The PMCF plan should include:
- All limitations in clinical data identified pre-market to be addressed.
- Justification as to how the PMCF activities will address these data limitations.
- Type of data to be generated in the post-market phase to further evaluate the device performance and safety.
- How these data will be generated in an appropriate time frame, including projections on the annual patients numbers, and pre-defined milestones on the periodic data assessment, where appropriate.
Additionally, the notified body should address the orphan device status of the device as early as possible before starting the conformity assessment. In the technical documentation review, the notified body should follow the same principle as for non-orphan medical devices. However, when assessing the clinical evaluation of orphan devices, the guidelines from the MDCG should be taken into account. The MDCG also addressees eventual involvement of expert panels to give advice on the device status and the clinical data needed for the clinical evaluation.
Lastly, the guidance includes three appendixes regarding:
- The information necessary for the clinical evaluation report of the “orphan devices”
- The different approaches and considerations that device developers could consider when designing a clinical investigation of an orphan device
- The extrapolation of clinical data to orphan indications
Have a look the whole guidance on clinical evaluation of orphan medical devices that you can find in our dedicated Library of Documents.
Do you have questions on clinical evaluation? Contact us here.
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