(161) Opportunities and Challenges for Decentralized Clinical Trials: The European Regulators' Perspective

Publication Number: 492

Background: Decentralized clinical trial (DCT) approaches – that center trial activities around the participant instead of the investigative site – have the potential to improve clinical trial conduct. However, there is limited information on the regulatory acceptance of these strategies.

Objectives: To identify opportunities and challenges that impact the implementation and authorization of DCTs from a regulators’ perspective.

Methods: Semi-structured interviews with European clinical regulators were conducted between May and October 2021. Regulators from 27 European countries were invited. The interview guide included topics on scientific and operational aspects of DCTs. Data were analyzed according to thematic analyses.

Results: Twenty regulators from 11 national competent authorities participated in one of the 15 interviews. Five major themes were identified from the interview data: justification, responsibilities, participant interests, data quality, and future perspectives. The respondents indicated that a scientific justification should underlie a DCT approach depending on the clinical trial context. Although all therapeutic areas may be considered for a DCT, several interviewees indicated that DCTs could be considered particularly in the context of rare diseases, chronic diseases, and low-risk clinical trials. In addition, participant safety and investigator oversight should be safeguarded when in-person visits are limited and when involving third parties. The respondents recognized the potential of DCTs to recruit participants who would not participate in a conventional clinical trial. Nevertheless, several regulators expressed concerns regarding generalizability, increase of missing data, increased variability in trial results, and validation of novel digital endpoints in the context of DCTs. The interviewees indicated that gaining experience with “decentralized elements” exerted by combining on-site and decentralized elements, and harmonization across Europe could facilitate the implementation of full DCTs in the future.

Conclusions: Key opportunities of DCTs include decreasing participation burden, allowing underserved groups to participate in CTs, and capturing data closer to the real-world. However, maintaining investigator oversight when involving third parties are challenges to the implementation of DCTs. The possible impact of decentralization on the data quality should furthermore be addressed when designing a DCT. Considering the factors identified in this study, the EU regulatory network is ready to gain experience with DCTs to make trials more participant-centered.