– Written by Anne-Sophie Bekx, PHUSE EU Connect Chair 2024 and Head of Submissions & Regulatory Intelligence at Johnson & Johnson and Jean-Marc Ferran, PHUSE Data Transparency Working Group Lead and Consultant & Owner at Qualiance

Introduction

The EMA ACT EU Clinical Trial Analytics workshop, held at the EMA Amsterdam offices on 25 and 26 January 2024, served as a platform for key stakeholders in clinical research to explore collaboration and innovation opportunities. The event, led by central figures including Peter Arlett, Lars Bo Nielsen from the DKMA (the Danish Medicines Agency) and Emer Cooke, focused on integrating data analytics into the clinical trial regulatory process. The discussions aimed to leverage the Clinical Trials Information System (CTIS), and other data resources, to enhance patient outcomes, moving beyond mere data collection to creating actionable insights.

This article provides our summary of the workshop, highlighting its objectives, participants, and the valuable discussions that took place.

Overview & Objectives

The EMA ACT EU Clinical Trial Analytics workshop gathered stakeholders from local regulatory agencies, national ethics committees, patient associations, pharmaceutical trade associations, funders, and academia, among others. This diverse gathering ensured multiple perspectives were considered, resulting in rich and comprehensive discussions.

ACT EU builds on the momentum generated by the Clinical Trials Regulation (CTR) and the recent launch of CTIS and aims to create a conducive environment for research and development in life sciences. By fostering harmonisation, innovation and collaboration, ACT EU strives to enhance the efficiency and effectiveness of clinical trials.

The primary objective of the workshop was to define use cases for data reuse considering both current EMA data sources (e.g. EudraCT, CTIS, CDP) and data not yet collected but deemed necessary, with the long-term aim of addressing the key question “How can we combine data to better develop evidence-based treatments?”

Role of PHUSE

PHUSE has been an EMA stakeholder since 2014, and our Working Group representatives are often invited to contribute to forums and workshops. The PHUSE Data Transparency Working Group has, over the years, interacted with the EMA and other agencies, such as Health Canada and the FDA, through commenting at times of public review of policies and guidance, participating in regulator-owned working groups (i.e. the EMA Technical Anonymization Group and the Health Canada PRCI stakeholders group) and hosting sessions with regulator presentations and panel discussions at conferences or at the CSS.

PHUSE was privileged to be invited as a pharmaceutical trade association, and Anne-Sophie Bekx (PHUSE EU Connect Chair 2024 and Head of Submissions & Regulatory Intelligence at Johnson & Johnson) and Jean-Marc Ferran (PHUSE Data Transparency Working Group Lead and Consultant & Owner at Qualiance) were thrilled to act as its spokespeople, providing expertise and perspectives from both the data standards and the data sharing sides.

Highlights

On the first day, participants across stakeholder groups presented their projects, challenges, opportunities, and ideas for improvement with regard to collection, availability and usage of clinical data published by the EMA (e.g. EudraCT, CTIS, CDP) or others (e.g. ClinicalTrials.gov). The presentations allowed for a better understanding of the current landscape and provided insights into areas for improvement.

Multiple stakeholders highlighted the potential and challenges of CTIS, emphasising the need for accurate and timely reporting to help feed downstream use of this database by patient organisation, regulator and funder. There is a real need to have precise and valid “data about clinical trials” to 1) know when a given question is being investigated in different countries to facilitate collaboration 2) identify a research gap where funders could reconsider their investments towards under-researched indications in comparison to their burden on society 3) investigate policy impact 4) enable patients to find clinical trials near them that they are eligible to participate in.

Day two was dedicated to breakout sessions, delving deeper into priorities from the perspective of 1) academia 2) ethics/HCPs/HTAs 3) data standardisation 4) industry and 5) patients. The following trends emerged across the groups:

• Learning from Failure: Whether it is the case of a study ending prematurely, or a study failing, or at a programme level when a submission is withdrawn or goes through a refusal, it is crucial this information is shared. Smaller companies would be reliant on investments and limited budgets. Learning from others’ failures seemed to be almost a matter of survival, and there was a wish to see terminated research programmes published right away. It was not clear, though, how such experiences and learnings could be efficiently shared, as this could be seen as sensitive information. More discussions and thought will be needed.
• Data Querying: In many instances, it was mentioned that an API to query CTIS data would be very helpful. This would mean that data in CTIS needs to be relevant, reliable, accurate and complete. Too often today, patient organisations are forced to build their own registries (when they can), but is this something they should be spending time and resources on, if it takes away from patient advocacy?
• Future Proofing: There is increasing demand for standardisation and harmonisation. There is an increase in demand to coordinate registries such as CTIS and ct.gov so that patients, academia, funders and others have a “single source of truth” when it comes to the landscape of available clinical trials today. This single source of truth argument also came up during the discussion on the interoperability of data. Standards, and controlled terminology in particular, were highlighted as being key in this.

Conclusion and Next Steps

The EMA ACT EU Clinical Trial Analytics workshop provided a platform for diverse stakeholders to come together, share insights and explore opportunities for collaboration and innovation. By addressing challenges in clinical data collection, availability and usage, participants demonstrated a shared commitment to improving the efficiency and effectiveness of clinical research. This workshop marks an important step forward in creating a more integrated, patient-focused and efficient clinical trial environment within the EU. The commitment to global standards and collaboration is expected to reduce duplicated research efforts and enhance patient involvement. While detailed next steps are still unclear at this point, the EMA will summarise the use cases defined as part of the breakout sessions on Day 2, with a report expected later in the year, and look into how to deliver on these use cases in the future.

Documents and recordings of the event are available on the EMA event page.