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Trials@Home: Center of Excellence – Remote Decentralised Clinical Trials

Periodic Reporting for period 2 - Trials@Home (Trials@Home: Center of Excellence – Remote Decentralised Clinical Trials)

Reporting period: 2020-09-01 to 2021-08-31

Clinical trials are crucial in drug development and improving patient health. However, they traditionally require participants to attend F2F study visits at clinical sites. While these trials produce valid results, they can bring a significant burden to participants and risk excluding people who are unable or unwilling to travel for study visits. (S)low patient recruitment and low retention put pressure on the efficiency, generalisability and validity of traditional, clinical site-centred trials.

Decentralised clinical trials (DCTs) hold the promise of solving this problem. The DCT approach is an operational strategy for technology-enhanced clinical trials, which are more accessible to patients by moving clinical trial activities to more local settings. Since this reduces the time spent attending study sites, DCTs have the potential to make taking part in a clinical trial more convenient.

The overall goal of Trials@Home is to reshape clinical trial design, conduct and operations, by developing and piloting standards, recommendations and tools for the definition and operationalisation of DCTs in Europe. By working together to share knowledge and experience, this consortium of public and private partners aims to improve the understanding and delivery of DCTs across Europe and worldwide.
Prior and ongoing experiences with DCTs have been analysed through case studies and interviews to provide best practices and selection criteria for DCTs and recommendations for implementation into RADIAL (Remote And Decentralised Innovative Approaches to cLinical trials), the T@H pan-EU pilot study. The guidance for all stakeholders on best practice regarding DCTs and criteria for selection of appropriate trials can be accessed on the project's website: T@H is in the process of updating the ongoing case studies and adding new case studies.

With regard to technologies, an elaborate technology scan was performed on technologies suitable for DCT approaches, concluded by an internal and external RFI. The results and the process of the technology scan activities have been published in “An Overview of Technology to Support Remote Decentralised Clinical Trials”. After this, an RFP was launched to select the technology package for RADIAL. Two internal technologies and over 30 external technologies were assessed, after which six providers were selected. This process is described in more detail in Deliverable 6.5 which is available on the website as well.
Additionally, a basic integration profile as well as an interoperability recommendation have been developed, which can already serve as blueprint for DCT architectures. This is planned to be published in a scientific journal or conference.

In the meantime, the preparations for RADIAL are in full swing. RADIAL has been set up as a parallel-group, open-label, multicenter study in Europe, which evaluates the scientific and operational quality of fully decentralized and hybrid approaches and evaluates the feasibility of such approaches compared to a conventional trial approach. All pivotal choices have been made for RADIAL: TA, primary endpoint(s), number of trial arms, randomisation strategy, sample size, IMP & intervention, country choice, study population, adaptive design options, and technology package. In addition, the final list of KPIs to be used to qualify and quantify the flow of activities in the RADIAL study has been determined. The draft protocol has been developed and was reviewed extensively by internal and external stakeholders, including patient representatives, regulatory bodies, health care providers, clinical trial specialists, technology experts, ethicists, legal experts, etc. In addition, the trial governance structure, which involves a quality workgroup and independent board, has been set-up and is now in function.

As part of Trials@Home, the regulatory, ethical, GCP, and legal aspects of the current EU environment relevant to DCTs will also be assessed and innovated where possible. To achieve this, several EU Member States were mapped, an internal survey on opportunities, barriers, and solutions for DCTs was set out, and semi-structured interviews with European regulators were conducted to identify regulatory challenges and opportunities that impact the authorisation and implementation of trials with remote elements. In addition, literature research on remote elements in recent phase 2-4 protocols, direct-to-patient IMP supply, and ethical aspects of DCTs is ongoing. Furthermore, a mock ethics review was set out to identify national competent authorities’/ethics committees’ opinions on RADIAL, in order to develop guidance for researchers and CAs/ECs on ethics review of DCTs.

With regard to stakeholder engagement, three surveys have been conducted to probe the knowledge on CTs and DCTs of patients, PIs, and GPs. This will provide insight in the training needs for DCTs. Work on DCT awareness scripts has also been initiated, which will be used for videos for the RADIAL website. In general, there is a big uptake from internal project partners and external stakeholders in registering for the newsletter.

In terms of project management, a strong governance structure is in place for the project, including a Scientific Advisory Board, External Stakeholder Platform, and Patient Expert Panel.
To link terminologies, Trials@Home published a glossary which brings together and clarifies the language used about DCTs: This work is continuing with an exploration of the various terms used for DCTs, highlighting the boundaries between them and, if necessary, proposing homogenization. The Trials@Home glossary work is already being used by other stakeholders in the industry.

The research performed so far formed the basis for publications in prominent peer reviewed journals: Coyle et al., Learning from remote decentralised clinical trial experiences (10.1111/bcp.15003) and De Jong et al., COVID-19 and the Emerging Regulatory Guidance for Ongoing Clinical Trials (10.1002/cpt.2225). Further publications are in development.

The Trials@Home consortium met with EMA’s ITF to discuss several aspects of the design of RADIAL. The interaction was successful and there was significant interest in the topic of DCTs, as demonstrated by 70+ participants from EMA’s side. The ITF group included 43 regulators from more than 15 National Competent Authorities from across Europe, which according to Dr Falk Ehmann, the ITF Chair, was an attendance record setting and reflected the great interest in DCTs. As follow-up to the ITF meeting, the consortium had an additional consultation with BfArM.

The Trials@Home team has also been active in several external communication activities, including e.g. webinars and conferences. The Trials@Home initiative is starting to be seen as a key initiative where new thinking and cross-industry alignment is starting to emerge to advance the DCT field forward in Europe.

The final Trials@Home DCT recommendations will be published in 2024. With further expected developments in technology and trials methods, the expertise will develop significantly over the next few years.
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