Trials@Home Annual General Meeting

Public Report

5-6 October 2020

September 2019, the great and grand kick-off meeting for the Trials@Home project in Paris.
A big project, a large group of people, some have left us, some have joined us in the meantime, but what a fun two days. We all went home feeling part of the group and ready to start working on this very challenging yet exiting project.

September 2020, due to the pandemic, a fully virtual annual meeting. A two-day virtual meeting with updates, insights, learnings and even a fully virtual, yet crazy, networking cocktail!

At the start of the meeting, we were all welcomed by the project leads Mira Zuidgeest (UMCU), Kim Hawkins (Sanofi) and Rick Grobbee (UMCU/Julius Clinical).

They, again, highlighted the importance of the project and complimented all partners on work already achieved and deliverables completed.

Rick Grobbee gave us an overview – based on survey results published in Nature Reviews Drug Discovery – on the current status of clinical trials. As we all know, a lot of trials have been suspended or delayed due to the COVID-19 outbreak and most new trials that are currently ongoing are pandemic-related. The investigators that were surveyed clearly indicated that telemedicine and remote patient monitoring were the one aspect of trials they were most interested in and that would bring the most value for future trials. These insights show that the world of clinical trials is ready for Remote Decentralised Clinical Trials (RDCTs) and thus Rick, again, pointed out the importance of this IMI project and the legacy it can leave behind for the future.

All work packages (WPs) were also given the opportunity to share progress and achieved goals with the consortium. What has happened and where are we now?

WP BEST created a long list of case studies to be looked at for best practices and already published their initial list of recommendations.

WP TECH went from high-level ideas to 7 Basic Building Blocks (BBBs) with concrete visions and “shopping lists” for the technology and services needed to roll out their envisioned RDCT. An internal and external Request for Information (RFI) were published and the assessment of these results is the base for the Open Call that will be published in the second year of the project to ensure that all the BBB technology and service requirements are available for the pilot study.

WP PILOT evolved from lively and lengthy discussions to an agreed study setup and investigational medicinal products (IMP) intervention. As previously announced in the first public webinar, the chosen therapeutic area for the pilot study will be type 2 diabetes.

WP EAGLE had a big challenge ahead of them at the start of the project as they needed to set up their cross WP function, start the country mapping, and draft their advice for EMA engagement. They succeeded!

WP CODE had the biggest impact on the project as they “created” the project window to the outside world by developing the project website, a public webinar, internal webinars, and a set of surveys aimed at probing different stakeholder groups about their training needs with regard to RDCTs.

Last but not least, WP PROMS – the glue between all the other WPs – went from having but a few names on a list to establishing and onboarding the External Stakeholder Panel (ESP), a body of external experts that will advise the WPs where needed, and the Scientific Advisory Board (SAB), an independent review organ that will be consulted only on specific deliverables. They also contributed, in close collaborations with TECH and CODE, on the open call for technology and services for the pilot study.

Apart from the plenary sessions where project updates were shared, there were also various breakout sessions on both days. An overview and highlights.

PILOT conducted an internal survey on the COVID-19 associated push on remoteness and how Trials@Home is adapting to improve execution in relation to this. The survey probed the consortium partners on the impact and consequences of COVID-19 on trial operations, associated problems and challenges, and the envisioned permanency of these changes. During breakout session dedicated to COVID-19 impact, the results of the survey were presented and discussed. The main takeaways were that some of the project partners will keep fully remote trials going forward, even if they had not done anything remote prior to the pandemic, and that telemedicine, IMP supply, and home health visits are the remote aspects that are most likely to be adopted permanently.

Maria Harrison, VP Real World Solutions and Digital Health from PRA Health Sciences, was invited to the session too and confirmed in her presentation that the pandemic has driven and accelerated innovation for clinical trials. She concluded by saying “the future ecosystem to conduct virtual trials is happening now and is here to stay.”

EAGLE organised a session focused on remote technologies, the ethical considerations behind them, and a live demo of the AiCure patient engagement platform.

Spencer Hay is the co-founder and chief scientific officer at Prism.Bio, an advanced analytics company that is developing tools to make the clinical trial data ecosystem more efficient and transparent. He presented some ethical considerations for the use of remote technologies in clinical trials and pointed out the remote tech and virtual trials resolve some, and amplify other, ethical concerns faced in site-based trials, while also transforming some familiar concerns into new challenges. He concluded his presentation saying it’s valuable to think of ethical concerns on a spectrum with important clinical trial factors and examine how the concepts relate to each other and highlighted that

“building trust through transparency will be key to the ethical use of these technologies.”

The second part of the EAGLE breakout session was headlined by Amanda Paley, Sr Director Clinical Development Strategy at AiCure. She focused her presentation on the importance of leveraging digital solutions to protect patient safety and trial data integrity, a feature embedded in AiCure´s patient engagement platform. Amanda also mentioned that compliance with global privacy regulations is key to scalability and that predictive modelling and other ‘smart’ means of patient engagement can improve resource allocation. She ended her presentation by emphasising that digital technology plays a crucial role in ensuring participant safety and oversight during decentralized trials and highlighted that

“the opportunity for low-friction collection of rich data sets presents novel opportunities.”. 

CODE and Lygature organised a joint session to explore what aspects and processes we can optimise and further improve within the project. What can be changed to the sharepoint My Project Plaza, new features, extra training? How can we improve internal communication, so that everybody gets the important stuff that is being shared and communicated without having to dig too deep or read too many emails and reports? And most importantly, what do we need to do differently in order to be able to reach out to people in other WPs more easily, to set up collaborations between WPs, and to get access to the available expertise within the consortium as soon as possible without having to jump through too many hurdles? These questions were debated in this session and clear recommendations were drafted and shared with the project management team.

We also wanted to hear from our valued ESP and SAB experts on the relevance of Trials@Home, so we invited Ole Dammann and Craig Lipset from the ESP and Peter Mol and Irene Schlünder from the SAB for an interactive Q&A. Rick Grobbee moderated the very lively debate.

TECH organised an inspiration session for attendees to listen and learn about other remote initiatives. First on was Cecile Spiertz, Senior Director External Innovation, Clinical Trial Platforms at Janssen, who is a project partner of EU-PEARL. Launched in November 2019, EU-PEARL aims to transform the future of drug development by creating a sustainable entity, co-designed by patients and available for industry and academia to conduct platform trials in any disease area.

The second presentation of the inspiration session was by Patrick Vandenberghe, Programme Officer at Electronic Components and Systems for European Leadership (ECSEL) Joint Undertaking. The ECSEL Joint Undertaking – a public-private partnership for Electronic Components and Systems – funds research, development and innovation projects for world-class expertise in these key enabling technologies, essential for Europe’s competitive leadership in the era of the digital economy. Through the ECSEL JU, the European industry, SMEs and research and technology organisations are supported and co-financed by 30 ECSEL participating states and the European Union.

Lastly, the floor was given to Bert Hartog, Senior Director Janssen Clinical Innovation at Johnson & Johnson to introduce us to Digital Endpoints Ecosystem and Platform (DEEP). Bart explained in great detail that despite the great advances in sensor and analytical technologies, the use of digital endpoints in regulatory submissions in clinical R&D is still very limited. Janssen Clinical Innovation has taken the initiative to launch an external strategic service design project to define a set of standard services that facilitate endpoint development and stimulate the ecosystem to accelerate the adoption of these tools and services. On the one hand, DEEP is an ecosystem of new harmonized and efficient services and on the other hand it’s a marketplace for collaboration and standardized and efficient asset trading.

[1] https://www.imi.europa.eu/projects-results/project-factsheets/eu-pearl

[2]

In the last breakout session, organised by UPPMD, it was all about the patient.

WPs TECH, BEST, EAGLE and PILOT were invited to highlight their views on where patient engagement is needed.

BEST started off by laying out that they have included patient perspectives in their systematic review inclusion criteria and extracted these as qualitative data for analysis. They have also interviewed patient representatives from two case studies and reached out to their local Public Involvement group for input in reviewing our draft recommendations. In the future, patients will be invited to suggest ways to solve issues encountered in their case studies and will be involved from the start of developing new sets of recommendations.

TECH described that very early on in the project they have set up a patient panel discussion to probe patients’ acceptance or reluctance with regard to RDCTs. From that two-hour discussion, user stories – short, simple descriptions of a feature told from the perspective of the person who desires the new capability (usually a user or customer of the system) – were extracted. An example was given: “As a patient I want to choose the time of medication delivery in order to have more flexibility.” These user stories are being used to develop the “shopping lists” for the various BBB technology packages.

PILOT has had discussions with UPPMD on how and when to involve ESP members, including patient representatives, in its pilot study. Going forward, when making design choices, PILOT has committed to keep in mind the user stories developed by TECH, as well as involve the International Diabetes Federation (IDF) Europe as therapeutic area-specific patient organisation with an active role in various working groups within the WP. And of course, patients will be involved in the dry run of the pilot study in the future.

Lastly, EAGLE informed us that IDF Europe and UPPMD are actively involved in the WP by attending various WP meetings and are thus consulted and informed on a regular base and their participation ensures that the patient voice is represented throughout all EAGLE outputs.

In conclusion:

The 2020 IMI Trials@Home virtual annual meeting was one full of information, updates, takeaways and conclusions. We had a great meeting with a full programme of interesting topics brought to us by experts from within the project and from outside.

We have to admit, we all missed the breaks where we could chat with other project partners about the project and progress of deliverables or even concoct new ideas. Still, despite the virtual aspect of the meeting, a lot of project partners did get to meet new people, if only through the quirky networking cocktail. Viva Variety!

Upwards and onwards to hopefully being able to hold a face-to-face meeting next September with more exciting news from the project and beyond.

All the slides and recorded session are available on MPP.

IMI
EFPIA
EU

This project has received funding from the Innovative Medicines Initiative 2 Joint Undertaking under grant agreement No 831458. This Joint Undertaking receives support from the European Union’s Horizon 2020 research and innovation programme and EFPIA.

Notes for editors – not for publication
If you would like more information, please contact the spokesperson at the UMC Utrecht.
Joris Prinssen: +31 6 2571 0234
press@umcutrecht.nl