RFPs: Facilitating the Conduct of Multinational Clinical Studies of Orphan Devices and/or of Highly Innovative Devices

Aims

• The topic aims at supporting activities that are enabling or contributing to one or several expected impacts of destination “Maintaining an innovative, sustainable, and competitive EU health industry”.

Scope

• The focus of this topic is on multinational clinical studies of orphan devices and/or of highly innovative (“breakthrough”) devices, including digital and Artificial Intelligence (AI) based tools and techniques.
• The emphasis within rare disease research and innovation has predominantly centred on pharmaceuticals, leaving a noticeable gap in the support for developing orphan devices. Orphan devices are specifically intended for use in rare diseases or conditions or in specific indications for rare cohorts of patients with an otherwise non-rare disease or condition. As, by their nature, orphan devices are intended for use in a small number of individuals each year, often infants and children, generating clinical data within an appropriate period of time and conducting clinical investigations is especially challenging due to low patient recruitment volumes.
• Besides orphan devices, also highly innovative (“breakthrough”) devices are in the scope of this topic if they are expected to provide major clinical benefits for the treatment, diagnosis or prevention of a life threatening, seriously debilitating or serious and chronic disease or condition, regardless of whether they target small patient populations. Highly innovative (“breakthrough”) devices aim to address unmet medical needs. ‘Unmet medical needs’ should be understood as a condition for which there exists no satisfactory method of diagnosis, prevention or treatment in the EU or, even if such a method exists, in relation to which the device concerned will be of major advantage to those affected. Those may include devices using digital tools and AI based technologies.
• Developers of such devices often face challenges to generate clinical data in the pre-market phase in a timely manner.
• Time and cost of clinical data collection can adversely affect public health by significantly delaying the availability of devices needed to treat or diagnose rare diseases or conditions or that may improve patient care or public health. Many devices are used off-label to respond to this unmet need. Nonetheless, a high level of clinical evidence based on thorough clinical data is needed to ensure patient safety.
• 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.

Funding Information

• Budget (EUR) – Year 2025: 40 000 000
• Contributions: 6000000 to 8000000

Expected Outcomes

• Proposals under this topic should aim to deliver results that are directed, tailored towards and contributing to all the following expected outcomes:
  • Healthcare providers increase their hands-on experience regarding the clinical use of orphan devices and/or of highly innovative (“breakthrough”) devices and get timely access to such devices with demonstrated clinical benefits;
  • Developers and manufacturers collect and obtain scientific evidence on their proposed intervention/ approach with the device under investigation;
  • Patients benefit from the development, studies and use of orphan devices and/or of highly innovative (“breakthrough”) devices;
  • Companies in the EU and associated countries get a better market position in this field and improve their knowledge on how to conduct multinational clinical studies for these devices.

Eligibility Criteria

• Entities eligible to participate:
  • Any legal entity, regardless of its place of establishment, including legal entities from nonassociated third countries or international organisations (including international European research organisations) is eligible to participate (whether it is eligible for funding or not), provided that the conditions laid down in the Horizon Europe Regulation have been met, along with any other conditions laid down in the specific call/topic.
  • A ‘legal entity’ means any natural or legal person created and recognised as such under national law, EU law or international law, which has legal personality and which may, acting in its own name, exercise rights and be subject to obligations, or an entity without legal personality.
• To become a beneficiary, legal entities must be eligible for funding.
• To be eligible for funding, applicants must be established in one of the following countries:
  • the Member States of the European Union, including their outermost regions:
    • Austria, Belgium, Bulgaria, Croatia, Cyprus, Czechia, Denmark, Estonia, Finland, France, Germany, Greece, Hungary, Ireland, Italy, Latvia, Lithuania, Luxembourg, Malta, Netherlands, Poland, Portugal, Romania, Slovakia, Slovenia, Spain, Sweden.
  • the Overseas Countries and Territories (OCTs) linked to the Member States:
    • Aruba (NL), Bonaire (NL), Curação (NL), French Polynesia (FR), French Southern and Antarctic Territories (FR), Greenland (DK), New Caledonia (FR), Saba (NL), Saint Barthélemy (FR), Sint Eustatius (NL), Sint Maarten (NL), St. Pierre and Miquelon (FR), Wallis and Futuna Islands (FR).
  • countries associated to Horizon Europe;
    • Albania, Armenia, Bosnia and Herzegovina, Canada, Faroe Islands, Georgia, Iceland, Israel, Kosovo, Moldova, Montenegro, New Zealand, North Macedonia, Norway, Serbia, Tunisia, Türkiye, Ukraine, United Kingdom.

For more information, visit EC.