Setting up a harmonised methodology to promote uptake of early feasibility studies for clinical and innovation excellence in the European Union

By setting up a harmonised EU methodology to promote the uptake of early feasibility studies (EFS)¹, this topic will improve patients’ access to health technologies, including digital technologies, support technological innovation, and contribute to a smoother development process for these health technologies. As such it will contribute to all the following IHI scientific, technological and economic expected impacts:

• Improve quality of clinical evidence on health technology innovation generated through earlier clinical experience obtained in the development process from an EFS.
• Facilitate uptake of early feasibility studies in health technology development, including for digital technologies.
• Increase the attractiveness of conducting clinical research and trials for healthcare technologies in the EU, including for SMEs, spin-offs and start-ups.
• Enable faster translation of health technology innovation into practice with increased access to treatment for patients, especially those with medical conditions that have limited or no alternative therapeutic options.

Better refined patient populations, their carers or patient representatives, and strengthened understanding of disease management and functional impairments, and treatment options.

^{1 Early feasibility studies are specified in the Questions & Answers Guidance from the Medical Device Coordination Group of April 2021: https://ec.europa.eu/health/system/files/2021-04/mdcg_2021-6_en_0.pdf}

The research and innovation action to be supported under this topic is expected to deliver results including a methodology for EFS in the EU, to facilitate compliance with the relevant legislation applicable in the EU, and a stakeholder network. It will contribute to all of the following expected outcomes:

• Patients and/or their representatives are engaged and contribute from the start of the development process of innovative health technologies.
• EU-wide and national regulators, health technologies assessment (HTA) bodies and notified bodies benefit from novel and robust methodologies, gain early knowledge on innovations, and can better anticipate and plan conformity assessment processes.
• Researchers, healthcare professionals, medical societies, and hospitals:
• contribute to the early generation of quality data;
• strengthen our understanding of disease management and treatment options that could inform future medical guideline development;
• provide input on innovation development;
• take part in the development of “hubs of clinical excellence”, thereby attracting investment into existing research and innovation as well as other areas (spin-off technologies).
• Health technology developers including those developing medical devices, drug-device combination products, imaging equipment and in-vitro diagnostics as well as SMEs, will have:
  • a controlled opportunity to assess their technologies and develop methods and best practices to support them in designing and conducting EFS when relevant.
  • early insights into the technology concept, patient characteristics and human factors that may impact technological performance, technology safety, future technological modifications or operator technique refinements.
  • a framework that will help to inform the subsequent development phase. In particular it will aid designing higher quality clinical studies while mitigating future patient risk, at the same time facilitating the conduct of future clinical investigations in broader patient populations.
• For SMEs, particularly, having access to a methodology and stakeholder network can facilitate the conduct of early feasibility studies. The availability of high-quality data early in the health technology development process would further support investment and development decisions.

The incremental development of innovative / breakthrough health technologies takes a long time, during which an innovation will have to successfully go through a process of testing and evidence generation before it can be launched.

As part of this process, early feasibility studies provide the opportunity to capture relevant additional information for the intended use from the real-world setting that would not be possible in non-clinical studies (i.e. bench testing and animal studies) at a very early stage. EFS can make it possible to optimise design and gain necessary information before running a large clinical investigation.

Even if it is legally possible to undertake EFS in the EU, such studies are not yet widely used. Indeed, most EFS are run today outside of the EU, and primarily in the United States¹.

This means that the EU may be at risk of losing out on an important opportunity to attract clinical research and further investments in innovation development to the region.

This topic seeks to develop and validate a methodology for EFS that is compliant with EU regulations, including a working methodology, easily accessible online, with information on how to undertake such studies, the process and requirements to follow and fulfil.

It also aims to bring together the relevant stakeholders that could have an interest in EFS and to facilitate use-cases where technologies would run the newly developed EFS methodological framework in order to test it and recommend any adjustments to be made to the methodology.

The project would entail the following:

• Research & analysis, including a review of existing international, EU and national guidelines, standards and best practice experiences. This would also include a survey of potential current gaps, barriers and challenges to undertaking EFS in the EU, taking into account the interplay between the different relevant current and future EU regulations.
• Development of an EU methodology for EFS
  • The methodological framework would include:i) definition and scope, including legal considerations;ii) the place of EFS in the development pathway of health technologies and when there is an added value for EFS;iii) the type of data required to conduct an EFS (technical data, preclinical data, number of patients, etc.);iv) process evaluation, methods and tools, including statistical tools adapted to the analysis of EFS results, and tailored to the needs and specificities of different health technologies, including digital and mobile health technologies;v) the contribution of EFS to making more patient-centred devices;vi) the contribution of EFS to the development of training plans for healthcare professionals that would in turn improve the use of devices.
  • Recommendations for best practices, addressing also ethical aspects from the outset, and contractual elements.
  • Development of a sustainable, freely-accessible online portal, hosted and maintained by the consortium, which would act as a repository for the methodological framework and the best practices, and which would facilitate interactions between stakeholders with an interest in EFS.
• Facilitate the creation of a sustainable stakeholder network at national and EU level
  • The network would promote the conduct of EFS and continue to gather experience from subsequent studies where appropriate and relevant to inform the EU EFS methodology.
  • Target groups include patient organisations and representatives, healthcare professionals, research institutions and hospitals, health technology developers, including SMEs, regulators, and HTA bodies.
• The selection of dedicated use-cases to inform, refine and validate the framework
  • The purpose of selected use-case technologies will be to undertake an EFS in the EU, whilst applying the methodology developed by the selected project, in order to test the methodological framework and evaluate the benefits for the conformity assessment process and patient access.
  • Learnings acquired on the use-cases will be used to adapt and finalise the methodological framework, and, where necessary, the blueprints and templates.
  • During the project execution, the consortium will define specific criteria and processes to determine which use-cases can be selected. Indicators of success will be developed and defined within each pilot trial, to compare it to other trials, and used as potential stop criteria.

^{1 US National Library of Medicine, ClinicalTrials.gov. Out of the 300 EFS referenced, only 8 are conducted in Europe as per September 2020.}