Summary: This descriptive methods paper reports how patient and public involvement and engagement (PPIE) shaped the design of ASCEND PLUS, a UK randomised controlled trial of oral semaglutide for the primary prevention of cardiovascular events in around 20,000 adults with type 2 diabetes. Six PPIE focus groups and a public advisory group informed a decentralised, direct-to-participant model with no physical sites. No cardiovascular or efficacy outcomes are reported, and no statistical testing was performed.
PICO Summary
| Element | Detail |
|---|---|
| Population | PPIE contributors (mainly UK adults living with type 2 diabetes) recruited to six focus groups and a public advisory group during the design of ASCEND PLUS, a decentralised RCT targeting around 20,000 participants in the UK. Descriptive, single-trial PPIE report. |
| Intervention | Coordinated PPIE programme: six focus groups plus a public advisory group, two members on the trial Steering Committee, two hybrid workshops, and review of participant-facing materials, online screening forms, videos, the trial website, and quality-of-life instruments. |
| Comparison | No formal comparator arm. Reflections are framed against conventional site-based trial design; this is a qualitative process report, not a controlled comparison. |
| Outcome | No effect sizes, confidence intervals, p values, ARR, or NNT (none applicable). Reported as themes: PPIE supported a choice between independent online and nurse-assisted screening/consent, a concise initial information leaflet, clearer public-facing materials, and input on quality-of-life instruments. Authors judge PPIE critical to securing regulatory and ethical approval. The cardiovascular outcomes trial remains ongoing. |
Expert Commentary
This paper should be read for what it is: a candid, descriptive account of how patient and public involvement was embedded into the design of a large decentralised trial, not evidence that oral semaglutide prevents cardiovascular events. The ASCEND PLUS efficacy results do not yet exist, and nothing here speaks to whether the drug works. Judged on its own terms, the report is useful. The decentralised, direct-to-participant model with no physical sites is operationally ambitious, and the described mechanisms, offering a choice between independent online and nurse-assisted consent, simplifying information leaflets, and co-reviewing public-facing materials, are plausible levers for accessibility and retention. The central limitation is that benefit is asserted rather than measured: with no comparator, no quantitative endpoints, and no significance testing, claims that PPIE was critical to approval and to trial quality rest on author reflection and cannot be verified from these data. Selection of engaged contributors may also overstate generalisability. Can I use this with my patients? Not directly, since this is a trial-design report rather than a clinical finding, though it is a worthwhile template for any team planning inclusive, decentralised research. Readers should also note that ASCEND PLUS evaluates a manufacturer-relevant product, so the parent trial’s sponsorship warrants scrutiny when its outcomes eventually report. Teams designing decentralised trials should treat this as a practical checklist while awaiting the hard endpoints.
References
El-Nayir M, Wijesurendra R, Preiss D, et al. Patient and public involvement and engagement in the ASCEND PLUS trial: reflections from the design of a streamlined and decentralised clinical trial. Trials. 2024;25(1):554. doi:10.1186/s13063-024-08393-2
