Accelerating Clinical Trials in the EU (ACT EU): Focus on Decentralized Clinical Trial Innovation

This blog summarizes the Accelerating Clinical Trials in the EU (ACT EU) initiative and discusses the first stakeholder meeting at the European Medicines Agency (EMA) on the development of a harmonized decentralized clinical trials (DCT) recommendation paper. Medidata representatives were at the in-person meeting and presented their views as experts in clinical trial technology.

Introduction to ACT EU

Advances in science and clinical trial technology have facilitated innovation and transformed all areas of the clinical trial sector and healthcare system. The ACT EU initiative was set up to further promote this innovation. ACT EU was launched in January 2022 and is co-led by the EMA, the European Commission (EC), and the Heads of Medicines Agency (HMA). 

The overarching EU Pharmaceutical Strategy and EU Network Strategy 2025 are foundational to ACT EU. These strategies aim to future-proof the current regulatory framework, supporting industry in promoting research and leveraging decentralized technologies.

ACT EU specifically aims to transform how clinical trials are designed, conducted, and served to strengthen the EU environment for clinical trial conduct and promote integration of clinical research in the European health system—all while maintaining the highest level of clinical trial data protection, robustness, and transparency. 

The ACT EU strategy has ambitious goals for innovation. It outlines ten priority actions to provide an overall, multi-stakeholder platform approach to clinical trials, as well as a modernization and acceleration of clinical research in the EU. One action is focused on methodology guidelines and the development of EU recommendations on conducting DCTs will be a key outcome and deliverable. 

Multi-Stakeholder Workshop on Decentralized Clinical Trials

In October 2022, the ACT EU initiative hosted their first multi-stakeholder workshop on decentralized trials at the EMA in Amsterdam. This meeting brought together expert stakeholders representing regulators, sponsors, CROs, patients, and clinical trial sites to share experiences and perspectives on DCTs. Medidata had the opportunity to attend in person, representing the Association of Clinical Research Organizations (ACRO) and the European CRO Federation (EUCROF), alongside the EU regulatory authorities, the European Federation of Pharmaceutical Industries and Associations (EFPIA), the European Organisation for Research and Treatment of Cancer (EORTC) and other patient and site representatives.

The EU DCT Recommendations Paper: A Regulatory Overview

The regulatory representatives kicked off the meeting with an overview of ACT EU and the scope and plan for the EU DCT Recommendations Paper. Version 1.0 of the paper is due to be published at the end of 2022. It was explained that the document would be dynamic and evolve through 2023. Aspects of clinical trial data protection and patient data privacy will be addressed in a latter version. The initial scope will include eight sections as outlined:

• Introducing a risk-based approach, which will speak to the overall landscape of DCT components and the continued importance of compliance to good clinical practice and applicable national laws, regulations, and standards.

• A look into the considerations and obligations of the sponsor and investigator—particularly when deploying a DCT where other third parties are involved, such as technology vendors, home health nurses, and direct-to-patient couriers.

• Examining the use of informed consent, the methods of informing the participant, and the various ways of signing when adopting a DCT model.

• The delivery of medicinal products and administration at home—covering the considerations around the shipment of investigational drugs to participants and how the medication is stored at trial participants’ homes.

• A discussion on how a medicinal product is administered either by patients themselves or via home healthcare nurses or carers; the importance of flexibility and trust when it comes to the participant and study team relationship.

• Defining and handling source data—discussing the expectation that more data will be captured outside of the clinical trial site using multiple systems that should be fit for purpose and how all data collected should be credible, reliable, and verifiable.

• Looking at trial monitoring, remote source data verification and review, and centralized monitoring. 

• There will also be a key appendix with national provisions for each of the components in scope across the 27 EU countries. This is seen as a significant step forward in supporting the industry when confirming local country laws upon considering a DCT approach.

Other Stakeholder Perspectives

There was an opportunity to hear from a cross-section of stakeholders:

• EFPIA spoke to the opportunities and challenges of running DCTs from a sponsor’s point of view with reference to the Trials@Home program.
• EORTC discussed the academic operational practicalities of running oncology trials during the pandemic, plus some of the lessons learned from continuing trials more remotely and through global lockdowns. 
• EUCROF provided an EU CRO perspective, highlighting the importance of harmonization and consideration to data protection and privacy aspects. 
• Medidata, representing ACRO, provided an overview of the ACRO DCT toolkit that had been developed over the last few years, including the Quality by Design Manual, Risk Assessment, DCT Quick Reference Guide, Data Flow Maps, and Change Management Guide for DCTs. Medidata also discussed the need for greater harmonization and transparency of member state positions to DCTs—thus the news of a national provisions appendix across 27 member states is very welcome.
• The patient and trial site representatives spoke about the importance of patient and investigator relationships; if remote strategies are leveraged, then there is a need to have at least one or more face-to-face meetings during a trial, as well as a general need for continued trust-building and flexibility. The importance of communicating follow-on trial results and returning data to patients was also raised. 

The ACT EU initiative is seen as a hugely positive step. This program sets ambitious plans to accelerate innovation and to further optimize the EU environment for clinical research, while maintaining high-level participant protection, data robustness, and transparency. 

The forthcoming EU DCT Recommendations paper will provide valuable insights to the sector and help build confidence in conducting DCTs. The collaboration on this topic across the multiple stakeholder groups has been impressive. Medidata looks forward to furthering the discussion and continuing to drive innovation in clinical research.