Who Should Decide the Outcome for a Clinical Trial? Comparing the Views of Stakeholders on Intervention Benefit Using...

Trials rarely include outcomes co-developed with stakeholders, which creates uncertainty about whether trial endpoints reflect the benefits valued by patients and clinicians. There is therefore a need to explore how stakeholder perspectives might influence the interpretation of trial results.

In a 2-phase study, we first engaged service users and staff to rank outcomes from a recently completed randomized controlled trial of cognitive remediation therapy (CRT). We then used multi-criteria decision modelling to generate value-weighted composite scores and reanalyzed the trial data from three arms: individual CRT, group CRT, and treatment as usual (TAU). This approach allowed us to examine how weighting outcomes differently, according to stakeholder perspectives, might affect the conclusions drawn from the trial.

Both stakeholder groups prioritized the Global Assessment Scale (primary outcome) and quality of life, but disagreed on the importance of cognition. Reanalyses using weighted composite scores produced the same robust finding as the original trial: CRT delivered in group or one-to-one formats provides significant benefit compared with TAU. Sensitivity analyses applying different stakeholder weights showed the same pattern. However, exploratory analyses revealed that group treatment for the service user weighted composite was not significant.

This study shows how stakeholder values can identify nuances in outcomes depending on the value placed on treatment benefits. Embedding this approach into trial design can strengthen the alignment of outcomes with patient and service priorities and help close the gap between randomized trials and service implementation.