Patient and Caregiver Perspectives on the Design and Execution of ACT-GLOBAL, a Stroke Adaptive Platform Trial: A Focus Group Study.

Introduction: Adaptive platform trials represent a paradigm shift in stroke research. We examined how patient-partners perceived the design and execution of an international platform trial in acute stroke, ACT-GLOBAL (A multi-faCtorial, mulTi-arm, multi-staGe, randomized, gLOBal Adaptive pLatform trial for stroke), through a series of focus groups with process evaluation methodology.

Methods: Participants were recruited from two comprehensive stroke centers one in Calgary, Canada and one in Sydney, Australia. Four virtual focus groups were attended by a total of 21 patient-partners and 11 clinician-researchers. One focus group had repeat attendees to review a draft consent form and patient information sheet, and one presented a video describing platform trials. Physician facilitators presented the platform trial concept followed by a facilitated discussion. Audio recordings were transcribed and combined with field notes. Exemplar quotes and themes were identified separately for each group and subsequently across groups.

Results: Patients/caregivers perceived acute stroke-focused adaptive platform trials such as ACT-GLOBAL as providing potentially beneficial opportunities to be randomized to multiple treatments, with efficiencies and richer data to improve patient care. Emphasis was given to the importance of gatekeeper processes for the addition of future questions posed by the platform to ensure examined questions would not interfere with routine care, and that safety decisions were ultimately made by nonconflicted parties. They appreciated that deferral of consent would be ideal to allow timely randomization/treatment within the adaptive stroke platform, and for patient safety to be prioritized in enrolment-related decisions with family input whenever feasible. The need to have trial information accessible in digestible chunks, multiple languages, and modalities was emphasized. To facilitate engagement, transparency, trust, and two-way communication was deemed critical to the informed consent process.

Conclusion: Patient-partners were supportive of an adaptive platform trial design. However, they expressed important priorities in their execution while safeguarding patient autonomy and safety. Oversight, multiple modes of delivering patient information, and having feedback evaluation and transparency with ongoing participation were identified as valued components. Deferral of consent was recognized as a pragmatic way to enroll patients. Similar considerations may apply to adaptive platform trials in other neurological/medical emergencies.