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

Speaker(s)

de Jong A1, Shahid N1, Zuidgeest M2, Santa-Ana-Tellez Y1, Hogervorst M1, Goettsch W1, Traore H3, de Boer A1, Gardarsdottir H1
1Utrecht University, Utrecht, UT, Netherlands, 2University Medical Center Utrecht, Julius Center, Utrecht, Utrecht, Netherlands, 3UCB Biopharma, Brussels, Brussels, Belgium

Presentation Documents

OBJECTIVES: Decentralized clincial trial (DCT) approaches move trial activities from traditional investigative sites to the trial participants’ direct surroundings. This study aimed to identify the opportunities and challenges for DCT approaches from an European health technology assessment (HTA) perspective.

METHODS: We conducted semi-structured interviews from September 2022 to February 2023 with representatives from European HTA bodies including pharmacotherapeutic assessors and pharmacoeconomic assessors. Interview transcripts were analyzed through thematic analysis.

RESULTS: In total, 25 respondents participated in one of the 24 interviews. Although the respondents had experience with individual DCT “elements” (e.g., ePROs and wearables), they did not have experience with assessing (full) DCTs and various respondents had not heard of DCTs before the interview. Two main themes were identified from the data: (i) the role of DCTs in HTA decision-making and (ii) the acceptability and relevance of data generated in a DCT. The first theme talks to the perceived suitability of DCTs and their role within the evidence framework. Interviewees mentioned that DCTs could be particularly suitable for chronic conditions for which validated (digital) endpoints are available. The respondents indicated that DCTs should be considered complementary to conventional randomized controlled trials (RCTs) and several respondents associated DCT approaches with an observational study design. As regards the second theme, the respondents expected DCTs to reduce recall bias and the results to be more representative of the real world because participants may not feel observed and a more diverse population may be recruited. However, the respondents mentioned that increased responsibility of participants in the data collection could jeopardize data quality.

CONCLUSIONS: European HTA representatives see opportunities for DCTs, including data collection in the real-world setting and the recruitment of more diverse participants. Increased awareness of DCTs including the identified opportunities and challenges could help HTA assessors in their appraisal of DCT approaches in the future.

Code

SA79

Topic

Medical Technologies, Study Approaches

Topic Subcategory

Clinical Trials

Disease

No Additional Disease & Conditions/Specialized Treatment Areas