Plenary Session: Pragmatic Trials—Bridging Research and Real-World Care

While explanatory clinical trials, which focus on evaluating therapeutic efficacy under ideal circumstances, are crucial for learning about new therapeutic interventions, they often include non-representative patients due to stringent eligibility criteria. Additionally, they often use intermediate endpoints, which can lead to uncertainty about the applicability of study results to patients in the real world.  Therefore, they may not offer the best setup to address clinically meaningful daily clinical questions based on patient-relevant endpoints, highlighting the need for optimizing therapeutic strategies. This can be partly achieved by incorporating more pragmatic elements into cancer clinical trials conducted in real-life settings to enhance patient-centricity, efficiency, and the generalizability of evidence. This approach could also consider the interests and needs of stakeholders in healthcare, HTA agencies, clinicians, payers, and most importantly, patients and caregivers.  However, these more pragmatic trials, especially those addressing the overutilization of therapeutic interventions, are not without their challenges. Patient acceptability, trial designs and feasibility, as well as fitness for regulatory frameworks and, finally, financial resources to conduct such trials, represent some of the most concerning challenges.  The panel will attempt to disentangle the role, benefits, and challenges of pragmatic trials. How pragmatic trials could help re-engineer the process of development into access. Can pragmatic clinical trials be part of the solution to addressing disparities and inequalities in access to clinical trials, thereby bringing faster therapeutic progress to all?