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 the Glossary with more information.
.If you need any additional information don’t hesitate to reach out to [email protected]
Adaptive Pathways – A flexible regulatory approach as part of the European Medicines Agency’s (EMA) efforts to improve timely access for patients to new medicines, particularly those addressing unmet medical needs. Adaptive pathways is a scientific concept for medicine development and data generation which allows for early and progressive patient access to a medicine.
Benefit–Risk Assessment – The systematic analysis ang weighing of the clinical benefits of a device against its potential risks, when it is used in accordance with the intended purpose defined by the manufacturer.
CE Marking – A marking by which a manufacturer indicates that a device is inconformity with the applicable requirements set out in this Regulation and other applicable Union legislation providing for its affixing.
Clinical Evaluation – A systematic and planned process to continuously generate, collect, analyse and assess the clinical data pertaining to a device in order to verify the safety and performance, including clinical benefits, of the device when used as intended by the manufacturer.
Clinical Evaluation Plan (CEP) – Mandatory technical document under EU MDR 2017/745 that defines the plan for conducting the clinical evaluation of a medical device, including the objectives, methodology, and strategy for collecting and assessing clinical evidence. The CEP is a key instrument guiding the device’s clinical strategy and must address all applicable regulatory requirements, remain consistent with the technical documentation and the state of the art, and reflect the device’s intended purpose and expected patient benefits. It provides a structured framework for the collection, analysis, and continuous update of clinical data throughout the device lifecycle.
Clinical Evaluation Report (CER) – Document summarising the results of the clinical evaluation, including the analysis of clinical data and the benefit-risk assessment.
Clinical Investigation – Any systematic investigation involving one or more human subjects, undertaken to assess the safety and the performance of a device.
Competent Authority – A regulatory authority in the European Union.
EUDAMED – The European Database on Medical Devices used for device and actors’ registration, traceability, surveillance and vigilance activities, ensuring the regulatory transparency within the EU.
Equivalence Assessment – Systematic and documented evaluation comparing a device with a similar authorised “equivalent” device across technical, biological, and clinical characteristics, in order to justify the use of clinical data from that device in the clinical evaluation.
Expert Panel –The medical device expert panels help ensure the safety and performance of medical devices on the EU market. The expert panels also facilitate their timely availability for patients.
General Safety and Performance Requirements (GSPRs) – Fundamental safety and performance requirements that medical devices must meet before being placed on the EU market (Annex I MDR/IVDR).
Horizon Europe – The European Union’s research and innovation funding programme supporting scientific projects, including clinical studies for innovative or orphan medical devices.
Medical Device Coordination Group (MDCG) – Expert group established under Article 103 MDR that supports the consistent implementation of EU medical device legislation and issues regulatory guidance documents.
Notified Body – An accredited body that conducts conformity assessments for medical devices.
Performance Evaluation Plan (PEP) – Mandatory technical document under EU IVDR 2017/746 that defines the plan for conducting the performance evaluation of an in vitro diagnostic medical device, including the objectives, methodology, and strategy for collecting and assessing scientific validity, analytical performance, and clinical performance data. It provides a structured framework for the collection, analysis, and continuous update of performance data throughout the device lifecycle.
Performance Evaluation Report (PER) – Mandatory technical document under EU IVDR 2017/746 that compiles data to demonstrate scientific validity, analytical performance, and clinical performance.
Post Market Performance Follow-up (PMPF) – Mandatory, proactive IVDR (EU 2017/746) process for CE-marked in vitro diagnostic (IVD) devices. It involves continuously collecting real-world data to confirm safety, performance, and risk-benefit ratios throughout a device’s lifecycle.
Periodic Safety Update Report (PSUR) – Periodic safety update report summarising safety and performance data for medium- and high-risk devices (class IIa, IIb, III-MDR and class C and D-IVDR) as a result of the post-market surveillance plan together with a rationale and description of any preventive and corrective actions taken.
Post-Market Clinical Follow-up (PMCF) – A continuous process that updates the clinical evaluation and is addressed in the manufacturer’s post-market surveillance (PMS) plan. Through PMCF, the manufacturer proactively collects and evaluates clinical data from the use of CE-marked devices within their intended purpose, with the aim of confirming safety and performance throughout the expected lifetime of the device, ensuring the continued acceptability of identified risks, and detecting emerging risks based on real-world evidence.
Post-Market Surveillance (PMS) – All activities carried out by manufacturers in cooperation with other economic operators to institute and keep up to date a systematic procedure to proactively collect and review experience gained from devices they place on the market, make available on the market or put into service for the purpose of identifying any need to immediately apply any necessary corrective or preventive actions.
Real-World Data (RWD) – RWD are routinely collected data relating to patient health status or the delivery of health care from a variety of sources other than traditional clinical trials (e.g. claims databases, hospital data, electronic health records, registries, Mhealth data, etc.).
Real-World Evidence (RWE) – RWE is evidence derived from the analysis of Real-World Data (RWD).
Registry-Based Study – Investigation of a research question using the data collection infrastructure or patient population of one or several patient registries. A registry-based study may apply primary data collection in addition to secondary use of the existing data in the registry.