Learning from COVID-19 related trial adaptations to inform efficient trial design—a sequential mixed methods study

Abstract

Background Many clinical trial procedures were often undertaken in-person prior to the COVID-19 pandemic, which has resulted in adaptations to these procedures to enable trials to continue. The aim of this study was to understand whether the adaptations made to clinical trials by UK Clinical Trials Units (CTUs) during the pandemic have the potential to improve the efficiency of trials post-pandemic. Methods This was a mixed methods study, initially involving an online survey administered to all registered UK CTUs to identify studies that had made adaptations due to the pandemic. Representatives from selected studies were qualitatively interviewed to explore the adaptations made and their potential to improve the efficiency of future trials. A literature review was undertaken to locate published evidence concerning the investigated adaptations. The findings from the interviews were reviewed by a group of CTU and patient representatives within a workshop, where discussions focused on the potential of the adaptations to improve the efficiency of future trials. Results Forty studies were identified by the survey. Fourteen studies were selected and fifteen CTU staff were interviewed about the adaptations. The workshop included 15 CTU and 3 patient representatives. Adaptations were not seen as leading to direct efficiency savings for CTUs. However, three adaptations may have the potential to directly improve efficiencies for trial sites and participants beyond the pandemic: a split remote-first eligibility assessment, recruitment outside the NHS via a charity, and remote consent. There was a lack of published evidence to support the former two adaptations, however, remote consent is widely supported in the literature. Other identified adaptations may benefit by improving flexibility for the participant. Barriers to using these adaptations include the impact on scientific validity, limitations in the role of the CTU, and participant’s access to technology. Conclusions Three adaptations (a split remote-first eligibility assessment, recruitment outside the NHS via a charity, and remote consent) have the potential to improve clinical trials but only one (remote consent) is supported by evidence. These adaptations could be tested in future co-ordinated ‘studies within a trial’ (SWAT).

Effects of the COVID-19 Pandemic on SMA Screening and Care: Physician and Community Insights

“…For this reason, several neuromuscular disease research groups have recently published “roadmaps” outlining processes for developing and testing disease-specific assessment protocols and tools that enable remote care and for patients with neuromuscular diseases like SMA [ 43 – 45 ]. Furthermore, SMA researchers and clinicians concur that remote care and treatment plans should include refinement and validation of: Clinical outcome assessments, including assessments administered through telemedicine by a clinician and assessments made through patient reported outcome measures, for both motor and respiratory functions [ 29 , 43 , 46 ]. Local sites for the collection of blood samples, or mobile collection at patients’ homes [ 34 , 44 , 46 ].…”

“…Furthermore, SMA researchers and clinicians concur that remote care and treatment plans should include refinement and validation of: Clinical outcome assessments, including assessments administered through telemedicine by a clinician and assessments made through patient reported outcome measures, for both motor and respiratory functions [ 29 , 43 , 46 ]. Local sites for the collection of blood samples, or mobile collection at patients’ homes [ 34 , 44 , 46 ]. A plan for how to maintain the treatment administration schedule whenever possible while preserving patient safety [ 29 , 30 , 47 ].…”

“… Digital devices to monitor variables such as respiratory function, muscle strength and function, sleep, and physical activity [ 9 , 44 ]. For clinical trials, the development of additional remote protocols to enable digital recruitment, enrollment, and informed consent [ 7 , 9 , 46 ]. …”

“…Clinical outcome assessments, including assessments administered through telemedicine by a clinician and assessments made through patient reported outcome measures, for both motor and respiratory functions [ 29 , 43 , 46 ].…”

Impact of financial compensation on enrollment and participation in a remote, mobile-app based research study

“…By utilizing the MyCap study app, we were able to conduct this study in a fully remote environment. Since the onset of the COVID-19 pandemic, the literature indicates a growing number of studies incorporating remote aspects [32][33][34][35]. While there is evidence that remote data collection reduces the burden for participants [36] and makes studies more accessible [37], this does not mean there aren't obstacles or challenges for study teams to consider when designing a remote trial.…”