Presenters: Khadija Rantell (MHRA); Rima Izem (Novartis); Rackeh Williams (GSK), Luis Garcia-Gancedo (GSK); David Wright (AstraZeneca); Magnus Jörnten-Karlsson (Astra Zeneca); Magalie Hilton (roche); Yashoda Sharma (DiMe) 

In a decentralised clinical trial, patients’ assessments are (partly) transferred from the clinical trial site to the patients’ home and might occur without any face to face interactions between patient and study personnel. In particular during the Covid-19 pandemic, decentralising clinical trials attracted considerable attention as one way to continue conducting trials during lockdowns and curfews. In this online event, experts in the field discuss the opportunities and challenges of decentralised clinical trials for the development of drugs and the promise they hold for modernising the way trials are conducted.

The presentations in part 2 of this meeting were as follows:

Towards more patient-centric trials: opportunities and challenges of decentralized clinical trials. (Khadija Rantell)
The COVID-19 pandemic has necessitated flexibility in trial conduct and accelerated changes in the clinical trial landscape. Decentralised clinical trials (DCTs) introduce new approaches to the conduct of clinical trials that aim to make clinical trials more easily accessible and convenient for participants to take part in. Moving forward, it will be important to leverage the use of more innovative tools and approaches to trial design, and conduct, including adoption of remote approaches such as electronic patient-reported outcomes, home visits by health staff, follow-ups over phone, telemedicine etc. This talk will cover key considerations for suitability of use of decentralised clinical trial model, reflecting on the opportunities and challenges of implementing decentralised elements in clinical trials.

Decentralized clinical trials: scientific considerations through the lens of the estimand framework. (Rima Izem)
While the industry and regulators’ interest in decentralized methods is long-standing, the Covid-19 pandemic accelerated or broadened the adoption and the experience with these methods in clinical trials. Potential benefits of decentralizing studies include broadening access of clinical trials to a more diverse population, reducing the burden of participation in trials, or using innovative endpoints. As decentralization is moving from a necessity to a choice, researchers need to consider the actual value added and implications of these designs beyond the operational aspects of their implementation.
Our presentation illustrates how the estimand framework can clarify our thinking about those strategic decisions around decentralization. Those questions go beyond whether or not to decentralize, to how much and what to decentralize. That is, going through questions related to each attribute of the estimand, and especially population, treatment and variable, can guide the teams through a systematic thought process about the scientific opportunities, assumptions and potential risks associated with a possible use of decentralized components in the design of a trial. Illustrations from decentralized trial case studies will support the presentation and discussion of various considerations from this framework. Using this approach, teams can more easily calibrate the design decentralization complexity (from hybrid to fully decentralized) to the value added in answering scientific questions that could not be solely answered by an all on-site approach.

Developing Digital Biomarkers: Application to Rheumatoid Arthritis. (Rachel Williams and Luis Garcia-Gancedo)
Data collected in clinical trials present an incomplete view of disease activity due to daily symptoms fluctuation and heterogeneity. Digital technology can enable remote continuous monitoring and the addition of objective of patient assessments. Rheumatoid arthritis (RA) is a chronic condition with fluctuating symptoms allowing for feasibility assessment of digital biomarkers. First, we performed the PARADE study as the first in pharma to use the Apple Researchkit® for fully remote enrollment and data collection via the iPhone, including baseline information, symptoms, wrist movement, and a walk test. Based on this experience, we performed the weaRAble PRO study, enrolling patients through a clinic, and adding an Apple Watch as well as the iPhone for data collection. These studies indicate that digital technology allows for remote data collection, increased measurement frequency, a richer picture of disease activity, and a focus on patient centricity.

Panel discussion, with presenters from both days.
Discussion on the topics raised during the two half-days of the meeting, including input from the audience.