Regulatory science to support translational development of patient-centred health technologies

Expected Outcome

This 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”. To that end, proposals under this topic should aim to deliver results that are directed at, tailored towards and contributing to several of the following expected outcomes:

• Policymakers and regulators will get accelerated access to improved evidence driven methodologies to evaluate the impact and efficiency of novel health technologies, facilitating decision-making for their use in humans and uptake in clinical practice.
• Patients and the health systems will benefit from the more targeted and efficient uptake of safe and effective health innovations in clinical practice, supporting more personalised approaches and improved care and public health.

Scope

The development, uptake and impact of health technologies typically results from a long product development process that is based on a 'life cycle approach' which typically involves several iterations of defined stages, i.e. from development, assessment to post-market surveillance and post-market clinical follow-up.

While health technologies are governed by comprehensive legal frameworks aiming to ensure that health technologies are safe and effective, the regulatory science underlying these legal frameworks needs to be updated. This concerns inter alia i) more precise delineation of specific requirements (e.g. closing existing gaps concerning sufficiency of clinical evidence) and ii) the consideration of novel biomedical approaches, data and digital solutions (e.g. artificial intelligence - AI, virtual human twin, new approach methodologies as well as methods that cut through these domains) which model and predict relevant biological parameters and exploit relevant end-points and novel (bio)markers for clinical diagnostic and prognostic predictions. Such update of the regulatory science of health technologies should aim at supporting an effective adoption and uptake into routine use by health systems and end-users (healthcare providers, citizens), while maintaining guardrails to ensure that innovative health technologies are backed up by evidence of sufficient quality and relevance to the human situation.

Proposals can cover all types of health technologies, aiming to define improved and novel sources of evidence with proven relevance for regulatory decision-making with a focus on safety and performance throughout their lifecycle, i.e. throughout the continuous process of clinical evaluation. To this end, proposals should address either, or a combination of the following: i) the improvement of existing methodologies and their fitness to specific types or classes of health technologies, including methodology for regulatory assessment and ii) explore and examine to which extent novel information sources as indicated above can be considered as evidence that is satisfactory in view of regulatory needs concerning safety and performance.

Proposals should support the update and refinement of regulatory science on health technologies and contribute actionable information that can be used for improved or novel regulatory policies, rules, guidance documents and other tools with a view to ensuring that European patients and healthcare professionals have access to safe and effective innovative health technologies. Proposals should ultimately contribute to a regulatory environment that makes use of the full spectrum of novel biomedical and bio-digital approaches for clinical investigation and evaluation, while promoting a patient-centred approach to health technology innovation, facilitating the timely entry to market of performant and effective innovations and support their uptake in the health systems and clinical workflows without compromising patient safety.

Applicant consortia should reflect a broad representation of stakeholders, notably clinical societies, academia, notified bodies, industry, patients and regulators and the proposed work should address one or more of the following elements:

• Data and analyses on how existing approaches in regulatory science can be refined and improved in view of closing existing gaps of clarity, sufficiency of clinical evidence, generated on the basis of clinical studies and clinical investigations.
• Data and analyses on whether and to which extent novel information sources from biomedicine including new approach methods and digital and AI-enabled models and approaches can contribute to the clinical evaluation of innovative health technologies, e.g.: • By providing information on relevant biophysical, anatomical, physiological and other disease-relevant aspects.
• By supporting information integration through the use and aggregation of already existing data, including clinical ones, from similar types or groups of technologies (e.g. retrospective information in registries, data collections, including Real-World Data (RWD)[1] from using technologies that have characteristics that are relevant for innovative technologies).
• By supporting improved planning and design of first-in-man clinical studies, with a view of enhancing the effectiveness and the safety of such studies and rationalising the use of resources of all involved actors by focusing the generation and assessment of clinical data on health technologies for which those data are indispensable.

• Data and analyses that examine to which extent the above-mentioned points can support the development and uptake of innovative technologies for unmet medical needs and for special patient populations (e.g. paediatric and rare conditions) via dedicated regulatory pathways and/or within a structured framework enabling their development and testing in a real-world environment under regulatory supervision (“regulatory sandbox”).

The actual conduct of clinical studies[2] is not in scope of this topic.

The activities should cover and draw on all the relevant healthcare innovation related frameworks other than pharmaceutical products, i.e. medical devices, in-vitro diagnostics, AI, and Substances of Human Origin (SoHO).

The starting point is a good understanding of the innovative technology and of its inherent risks, so that appropriate safety and quality requirements can be applied for monitoring the outcome in the relevant healthcare setting. As the number of hybrid or combinations of health technologies increases and technology integration becomes rather the norm than an exception in health innovation, the current segregated, technology-specific, frameworks may not provide a clear path forward for the health technology that is targeted. To that end, when considering an innovation, it is important to consider all relevant legislative frameworks including MDR[3] and IVDR[4], the proposed SoHO-Regulation[5], and AI Act[6] among others.

Proposals are encouraged to consider, where relevant, the data, expertise and services offered by European research infrastructures especially those active in the health domain, such as EATRIS ERIC[7], and also the findings of previous EU-projects (e.g.: CORE-MD[8]).

[1] EMA definition: “Real-World Data are routinely collected data relating to patient health status or the delivery of healthcare from a variety of sources other than traditional clinical trials (e.g. claims databases, hospital data, electronic health records, registries, mhealth data, etc.)”.

[2] Please note that the definition of clinical studies (see introduction to this Work Programme part) is broad and it is recommended that you review it thoroughly before submitting your application.

[3] Regulation (EU) 2017/745 of the European Parliament and of the Council of 5 April 2017 on medical devices: https://eur-lex.europa.eu/eli/reg/2017/745/oj

[4] Regulation (EU) 2017/746 of the European Parliament and of the Council of 5 April 2017 on in vitro diagnostic medical devices: https://eur-lex.europa.eu/eli/reg/2017/746/oj

[5] Regulation (EU) 2024/1938 of the European Parliament and of the Council of 13 June 2024 on standards of quality and safety for substances of human origin intended for human application: https://eur-lex.europa.eu/eli/reg/2024/1938/oj

[6] https://digital-strategy.ec.europa.eu/en/policies/regulatory-framework-ai, https://eur-lex.europa.eu/eli/reg/2024/1689/oj

[7] European Infrastructure for Translational Medicine: https://www.eatris.eu

[8] Improved methods for clinical investigation and evaluation of high-risk medical devices: https://www.core-md.eu