Clinical development strategies for implementing multinational clinical studies have the potential to offer improved efficiency and to reach larger patient samples. Challenges may arise from the potential uncertainty regarding how regional disparities in regulatory, clinical, business, ethical and cultural practices may affect study design, conduct, data interpretation and various other outcomes.

This topic targets those challenges by supporting multinational studies aiming to gather pre- or post-market clinical data to demonstrate the device’s safety and performance (including determination of any undesirable side-effects and their acceptability when weighed against the expected clinical benefits).

The proposals should demonstrate that they address all the following activities for a device that is an orphan device or a highly innovative “breakthrough” device (or both), at any point of the pre-or post-market stage, including the development stage, with the overall purpose to generate data in support of CE marking under the Regulations on medical devices (MDR) or in vitro diagnostic medical devices (IVDR):

• Design and conduct multinational clinical studies in a minimum of two different countries in the EU or Associated Countries, with a focus on orphan devices and/or highly innovative (“breakthrough”) devices, with a view to demonstrate the safety and clinical performance of the device(s) subject to the study.
• Present a sound clinical study feasibility plan, including an appropriate patient selection and realistic recruitment plans at different sites, justified by scientific publications or preliminary results. Proposals should adopt a gender-sensitive and intersectional approach, considering individual characteristics such as gender, sex, race, ethnicity, disability and age. Additionally, socioeconomic, lifestyle and behavioural factors should be taken into account. For this, the topic requires the effective contribution of social sciences and humanities (SSH) disciplines and the involvement of SSH experts, institutions as well as the inclusion of relevant SSH expertise, in order to produce meaningful and significant effects enhancing the societal impact of the related research activities.
• Demonstrate potential clinical benefit for patients and healthcare providers, including quality of life and consideration of patient-reported outcomes when relevant.
• Involve patients, patient organisations, carers and healthcare professionals in the design of the clinical studies.
• Identify, collect and record relevant good practices and experiences related to the design, conduct, sample handling, data analysis and results reporting of multinational clinical studies. In addition, provide appropriate recommendations and lessons learned.
• For multinational clinical studies, authorisation for the study approval by more than one national competent authority may be necessary. Develop a regulatory strategy and interaction plan for generating appropriate evidence as well as engaging with regulators and other relevant bodies (e.g., European Medicines Agency (EMA), EMA expert panels, national regulators, Health Technology Assessment bodies, etc.) in a timely manner. Consider also the potential for future regulatory impact of the results.

Proposals may include multiple devices, but the minimum expected is one device.

Participation of small and medium-sized enterprises (SMEs) is strongly encouraged.

For orphan devices or highly innovative devices relevant to rare disease patients, applicants should look for complementarities and potential synergies with actions implemented under ERDERA the co-funded European Partnership on Rare Diseases proposed under Horizon Europe, as well as synergies with actions implemented under the EU4Health programme.