ACT EU Multi-stakeholder meeting on Decentralised Clinical Trials

The EU decentralized clinical trials (DCT) project aims to address some of the opportunities and challenges of the new and innovative clinical trial designs and methodologies.

On October 4th, 2022, The ACT EU held a multi-stakeholder workshop on behalf of the EU DCT project which was hosted by the European Medicines Agency (EMA). The workshop brought together participants from all areas of the research community.

The aim of the workshop was to get an overall picture of the EU DCT project group work, including the planned publication of a guidance paper, whilst gaining multi-stakeholder perspectives. Specifically targeting patient representatives and investigator site experts to produce concrete recommendations for the upcoming guidelines and the creation of a multi-stakeholder platform.

Besides the EU DCT project team, perspectives were incorporated from SAWP Scientific Advice Experts, MWP Data Methodology Experts, PDCO and Enpr- EMA Paediatric experts, PCWP Patient representatives, and HCPWP Health Care Professionals. 

• The biological activity of ATMPs can be mediated by numerous factors meaning one single marker or assay may not fully reflect the functionality of the product.
• Functional potency assays can be difficult to validate due to the cellular components and inherent variability related to the material and manufacturing process.
• Limited number of cells available in autologous therapies and needs for rapid patient administration (fresh products) may restrict potency testing possibilities at release

• The Authorities revealed the structure and the aim of the guidance paper; the roles, and responsibilities of the involved stakeholders in the DCT processes; the need for the establishment of an informed consent process; the importance of a detailed process of delivery of medical products, administration, and procedures at home; the intent to create awareness on the extensive data and to define and handle source data, while remote monitoring remains work in progress.
• The industry sponsors pinpointed that they see opportunities for DTC for enhancement of the research through DCT approaches in several aspects to reduce geographical barriers; the potential to increase diversity; reduce the burden of participants and caregivers; empower the participants with choice and facilitate innovation. The challenges span from the operational and technological; the relationship involved, to data quality and privacy, and the lack of regulatory guidance and framework.
   
• The academic sponsor gave a perspective on the experiences of the use of DCT elements during covid-19. The main point was (at least in oncology), that they are not ready for fully remote procedures, but rather hybrid forms. The decentralized processes currently come on top of the existing ones, and what is paramount, is that they require different setups in different EU member states.
• CRO insights and experiences revolved around the way to solve identified challenges in the implementation of DCT elements in clinical research. The three main points of consideration discussed were: patient safety; data protection; and quality of data (needed for optimal harmonization); as well as the site readiness for DTC elements (both logistically and technologically).

        Enabling transparency of Member State requirements and the development of a central repository of            national legislation, regulation, and guidance would mitigate greater coordination across regulatory agencies and would help advance DTC. 

• Patient Perspective: Hybrid solutions were the main point of discussion for patient representatives, enhancing the importance of having a choice. They discovered that decentralization could lower motivation if forced, and it can be an issue for people with low technical skills. The patient must own the data, and the data should be returned to the patient once the CT is completed. During the panel discussion the relationship, trust, co creation, and flexibility of the processes arose as keynotes. All panelists agreed that face-to-face interaction is still very important, at least in the initial phase. 
• The investigator representative argued that DCT is quite a new modality and DC elements are still a small percentage of studies and trials. DCT are evolving and are an important step in the field of clinical research. They are particularly useful for patients with rare diseases, patients with limited mobility, and/or functional disabilities that are not autonomous. The balance between the patient's needs and the healthcare professional’s workflow is paramount. Identification of adequate and secure electronic platforms as well as training is also needed. 

The world of CTs is rapidly changing, and DCT is here to stay. The experiences pinpoint the main goal – a better regulative framework, a harmonization on the EU level to facilitate large CTs and to develop the EU further as a competitive center for innovative clinical research.

The main logistical challenges leveraged the point for the need for balance between patient needs and the investigator; the possible hybrid models; the right technological development and training; data protection harmonization; and “one model does not fit all.”

The shared perspectives will serve as a foundation for the guidance paper planned for Q4, 2022 and the multi-stakeholder platform planned for 2023.