Periodic Reporting for period 5 - Trials@Home (Trials@Home: Center of Excellence – Remote Decentralised Clinical Trials)
Reporting period: 2023-09-01 to 2024-08-31
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, site-centred clinical trials.
Decentralised clinical trial (DCT) approaches hold the promise of solving this problem. The DCT approach is an operational model of clinical trials in which trial activities are designed to take place at, or in the vicinity of, the participant's home, rather than at a clinical site. This approach can make use of technologies and other innovative operational approaches to facilitate data collection. 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 DCT approaches 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 DCT approaches across Europe and worldwide.
Decentralised clinical trial (DCT) approaches hold the promise of solving this problem. The DCT approach is an operational model of clinical trials in which trial activities are designed to take place at, or in the vicinity of, the participant's home, rather than at a clinical site. This approach can make use of technologies and other innovative operational approaches to facilitate data collection. 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 DCT approaches 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 DCT approaches across Europe and worldwide.
Trials@Home is a collaborative initiative that brings together academic partners, SMEs, private foundations, patient organisations, and EFPIA partners, along with stakeholders from various fields such as medical, technological, regulatory, ethical, and social domains. The project aims to develop practical recommendations to support the widespread acceptance and use of decentralised clinical trial (DCT) approaches in Europe.
In its fifth year, RADIAL, the proof-of-concept study, achieved a significant milestone by recruiting its first participant on August 22, 2023, marking the transition from preparation to execution. To enhance recruitment efficiency, a substantial protocol amendment was submitted and approved in all participating countries, adjusting the inclusion and exclusion criteria. Recruitment continued until May 14, 2024, enrolling 100 participants in Part A and 8 in Part B.
The technology package for RADIAL was meticulously assembled, tested, and implemented. This included a comprehensive helpdesk system featuring a knowledge base and a ticketing system to streamline support requests and issue resolution. Ongoing manual data cleaning and automated query closure were performed to maintain data quality. The Data Management Plan (DMP) for RADIAL was developed and integrated into the overall project DMP. Detailed processes for the delivery of study medications and materials were established, ensuring smooth logistics. Extensive training materials for site staff and patients were developed and implemented, supported by the communication team and the Patient Expert Panel.
Research activities on best practices for DCTs continued with analysing the qualitative data collected from interviews on existing case studies related to DCTs. In addition, a database analysis was performed on the role of the physical examination in clinical trials and stakeholder interviews on this topic are planned. To analyse patient preferences, a discrete choice experiment has been set up, after an initial assessment on drivers and perceptions regarding participation in DCTs, for which a paper has been submitted to a peer-reviewed journal. Finally, several new publications on the ethical, regulatory, and operational challenges of DCTs have been published.
The consortium actively engaged with stakeholders through conferences, workshops, and webinars, disseminating findings and learnings from the Trials@Home project. The External Stakeholder Platform (ESP) and Patient Expert Panel (PEP) provided valuable input, ensuring the patient voice was reflected in the research process. A new team has been put together to start working on layman translations of papers and other documents produced by the consortium. The glossary team has also completed the final glossary update and collaborates on the alignment with other DCT-related glossary owners to ensure DCT terminologies are consistent across the European and American forums. The Sustainability Working Group focused on developing plans for the long-term sustainability of the project’s outputs, including collaboration with other initiatives and the development of course materials. A working group was also formed to focus on communicating about RADIAL outputs and learnings, starting with a newsletter distributed to sites.
Communication and dissemination activities were robust, with regular updates on the project website, social media, and newsletters. The project engaged with stakeholders through various events and developed training materials and awareness campaigns about DCTs. Overall, these activities generated valuable DCT knowledge, leading to the publication of scientific articles and public deliverables, all available via trialsathome.com.
In its fifth year, RADIAL, the proof-of-concept study, achieved a significant milestone by recruiting its first participant on August 22, 2023, marking the transition from preparation to execution. To enhance recruitment efficiency, a substantial protocol amendment was submitted and approved in all participating countries, adjusting the inclusion and exclusion criteria. Recruitment continued until May 14, 2024, enrolling 100 participants in Part A and 8 in Part B.
The technology package for RADIAL was meticulously assembled, tested, and implemented. This included a comprehensive helpdesk system featuring a knowledge base and a ticketing system to streamline support requests and issue resolution. Ongoing manual data cleaning and automated query closure were performed to maintain data quality. The Data Management Plan (DMP) for RADIAL was developed and integrated into the overall project DMP. Detailed processes for the delivery of study medications and materials were established, ensuring smooth logistics. Extensive training materials for site staff and patients were developed and implemented, supported by the communication team and the Patient Expert Panel.
Research activities on best practices for DCTs continued with analysing the qualitative data collected from interviews on existing case studies related to DCTs. In addition, a database analysis was performed on the role of the physical examination in clinical trials and stakeholder interviews on this topic are planned. To analyse patient preferences, a discrete choice experiment has been set up, after an initial assessment on drivers and perceptions regarding participation in DCTs, for which a paper has been submitted to a peer-reviewed journal. Finally, several new publications on the ethical, regulatory, and operational challenges of DCTs have been published.
The consortium actively engaged with stakeholders through conferences, workshops, and webinars, disseminating findings and learnings from the Trials@Home project. The External Stakeholder Platform (ESP) and Patient Expert Panel (PEP) provided valuable input, ensuring the patient voice was reflected in the research process. A new team has been put together to start working on layman translations of papers and other documents produced by the consortium. The glossary team has also completed the final glossary update and collaborates on the alignment with other DCT-related glossary owners to ensure DCT terminologies are consistent across the European and American forums. The Sustainability Working Group focused on developing plans for the long-term sustainability of the project’s outputs, including collaboration with other initiatives and the development of course materials. A working group was also formed to focus on communicating about RADIAL outputs and learnings, starting with a newsletter distributed to sites.
Communication and dissemination activities were robust, with regular updates on the project website, social media, and newsletters. The project engaged with stakeholders through various events and developed training materials and awareness campaigns about DCTs. Overall, these activities generated valuable DCT knowledge, leading to the publication of scientific articles and public deliverables, all available via trialsathome.com.
The Trials@Home initiative is a key platform where new thinking, cross-stakeholder alignment, and solid research on DCT approaches take place to inform and innovate within the DCT field in Europe. The research performed so far has formed the basis for 15 publications in prominent peer-reviewed journals as well as two PhD dissertations. The four articles published in RP5 address a wide breadth of topics within the DCT landscape, including EU HTA perspectives, ethical aspects, the soft impact of technology, and technological support systems, with many of these publications using RADIAL as a case study.
Beyond these publication efforts, the Trials@Home consortium is also actively involved in discussions around DCT approaches. Consortium members participated in 141 different events throughout the project (41 in year 5 alone), such as scientific conferences and regulatory meetings, to report on the impact of Trials@Home. The final Trials@Home DCT recommendations will be published at the end of the project. With further expected developments in technology and trial methods, the expertise and impact will further develop over the final months of the project. More scientific papers are expected, which are now in various stages of development. The Trials@Home initiative continues to be seen as a key platform where new thinking and cross-industry alignment are starting to emerge to advance the DCT field forward in Europe.
Beyond these publication efforts, the Trials@Home consortium is also actively involved in discussions around DCT approaches. Consortium members participated in 141 different events throughout the project (41 in year 5 alone), such as scientific conferences and regulatory meetings, to report on the impact of Trials@Home. The final Trials@Home DCT recommendations will be published at the end of the project. With further expected developments in technology and trial methods, the expertise and impact will further develop over the final months of the project. More scientific papers are expected, which are now in various stages of development. The Trials@Home initiative continues to be seen as a key platform where new thinking and cross-industry alignment are starting to emerge to advance the DCT field forward in Europe.