Opportunities and Challenges for Decentralized Clinical Trial Approaches: European Health Technology Assessment Perspective

Decentralized clinical trial (DCT) approaches are clinical trials in which some or all trial activities take place closer to participants’ proximities instead of a traditional investigative site. Data from DCTs may be used for clinical and economic evaluations by health technology assessment (HTA) bodies to support reimbursement decision making. This study aimed to explore the opportunities and challenges for DCT approaches from an HTA perspective by interviewing representatives from European HTA bodies. We conducted semistructured interviews with 25 European HTA representatives between September 2022 and February 2023, and transcripts were analyzed after thematic analysis. Two main themes were identified from the data relating to (1) DCT approaches in HTA and (2) trial-level acceptance and relevance. Experience with assessing DCTs was limited and a variety of knowledge about DCTs was observed. The respondents recognized the opportunity of DCTs to reduce recall bias when participant-reported outcome data can be collected more frequently and conveniently from home. Concerns were expressed about the data quality when participants become responsible for data collection. Despite this challenge, the respondents recognized the potential of DCTs to increase the generalizability of results because data can be collected in a setting reflective of the everyday situation potentially from a more diverse participant group. DCTs could generate relevant results for HTA decision making when data are collected in a real-world setting from a diverse participant group. Increased awareness of the opportunities and challenges could help HTA assessors in their appraisal of DCT approaches. •Decentralized clinical trial (DCT) approaches move trial activities from the investigative site to the participants’ surroundings, to render either fully decentralized trials or trials with a combination of at-home and in-clinic activities. These approaches have the potential to reduce participation burden, leverage technologies to collect data, and increase participant representativeness. In Europe, regulators are beginning to explore DCT approaches and have issued guidance documents, but the perspective of health technology assessors has not been studied before.•We identified opportunities and challenges for DCT approaches from a health technology assessment (HTA) perspective. The main opportunities relate to a better reflection of the real-world, for example, because participants feel less