2025 SCOPE Summit: Transforming Clinical Trials with Pragmatic Trial Approaches

The 2025 SCOPE Summit brought together industry leaders to explore integrating real-world data into clinical trials, focusing on pragmatic trials as a transformative approach. Key discussions revolved around regulatory initiatives, innovative trial designs, and operational strategies to enhance the applicability of clinical research in real-world settings. The summit highlighted the potential of pragmatic trials to bridge the gap between clinical research and practice, offering insights into how these trials can streamline processes, reduce burdens, and improve patient outcomes.

Regulatory Initiatives for Pragmatic Trials

The summit’s opening session, led by Gracy Crane from Roche Products Ltd, delved into the regulatory frameworks essential for supporting pragmatic trials. Crane emphasized the critical role of these frameworks in enabling trials to transition from controlled research environments to real-world clinical practice. The session highlighted the FDA’s efforts to integrate real-world data into clinical trials, addressing the effectiveness of therapies beyond the confines of traditional randomized controlled trials (RCTs). Crane pointed out that pragmatic trials are designed to answer whether a therapy works in diverse, real-world settings, a question often left unanswered by traditional RCTs due to their controlled nature and homogeneous populations. This regulatory focus aims to ensure that pragmatic trials can provide substantial evidence for drug approvals, making them a valuable tool for both sponsors and regulators.

The discussion also touched on the importance of policy drivers and the momentum required to sustain these innovative approaches. Crane noted that pragmatic trials offer flexibility, allowing for integrating existing data and enrolling diverse populations. This adaptability enhances the relevance and applicability of clinical research, ultimately bridging the gap between efficacy and effectiveness. By focusing on real-world applicability, pragmatic trials can provide the substantial evidence needed for regulatory approvals, thus playing a crucial role in the future of drug development.

The Role of Pragmatic Trials in Clinical Practice

Pragmatic trials are designed to make clinical trial results applicable to broader, real-world populations, addressing the gap between efficacy and effectiveness. The summit explored how these trials can serve as a bridge, ensuring that therapies are effective in diverse clinical settings. The discussion included the concept of the Pressi diagram, which outlines various domains of pragmatism in trials, such as eligibility criteria and trial settings. This diagram guides trial designers to select which pragmatic elements to incorporate, such as broadening inclusion criteria or shifting trial settings from academic centers to community clinics.

The flexibility offered by pragmatic trials was highlighted as a key factor in their potential to provide substantial evidence for drug approvals. This adaptability allows for integrating existing data and the enrollment of diverse populations, ultimately enhancing the relevance and applicability of clinical research. By focusing on real-world applicability, pragmatic trials can provide the substantial evidence needed for regulatory approvals, thus playing a crucial role in the future of drug development.

Innovation in Clinical Trial Design

Henry Wei from Regeneron Pharmaceuticals shared insights into the innovative approaches adopted in clinical trial design. Wei, who leads the Development Innovation group, emphasized the importance of collaboration and the role of organizations like Transcelerate in fostering innovation. He highlighted the need for trials that are efficient and capable of integrating seamlessly into clinical practice. Wei pointed out that the current healthcare landscape demands trials that reduce the burden on healthcare providers and improve the speed and quality of clinical research.

Wei mentioned the collaborative environment of Transcelerate as a “treehouse” where industry leaders can come together to share ideas and drive innovation. This collaborative approach is crucial for developing trials that are more streamlined and accessible, ultimately benefiting both patients and healthcare providers. By fostering a culture of innovation and collaboration, the industry can create more efficient trials reflective of real-world clinical practice, ultimately improving patient outcomes and advancing clinical research.

FDA’s Center for Clinical Trial Innovation

Kevin Bugin, formerly Deputy Director of Operations in the FDA’s Office of New Drugs and now at Amgen, stepped in for the originally scheduled FDA speaker to discuss the FDA’s Center for Clinical Trial Innovation (C3TI). He provided insights into the center’s demonstration programs, which aim to integrate pragmatic elements into clinical trials, such as streamlined trials embedded in routine clinical practice. These initiatives help sponsors collaborate more effectively with the FDA, supporting the broader adoption of innovative trial designs. Bugin emphasized the importance of these collaborations in ensuring the successful implementation of pragmatic trial approaches.

The C3TI’s initiatives, such as the Bayesian supplementary analyses and the collection of safety data, are intended to build experience and confidence in using these approaches. By partnering with sponsors, the FDA aims to learn from these trials and disseminate best practices across the industry, ultimately contributing to a more efficient and effective clinical trial ecosystem. These efforts are crucial for advancing clinical research and ensuring that trials are efficient and reflect real-world clinical practice.

Operational Considerations for Pragmatic Trials

Stephanie Derbyshire from Roche highlighted the operational challenges and considerations in implementing pragmatic trials. She stressed the need for a shift in thinking to accommodate the diverse patient populations and settings that pragmatic trials entail. Derbyshire discussed the importance of selecting the correct sites and patients and the need for tailored training and support for research-naive sites. She pointed out that traditional trial sites may not always reflect the diverse patient populations needed for pragmatic trials, necessitating the inclusion of community clinics and family practitioners.

This approach requires careful planning and support to ensure these sites can effectively participate in trials. Derbyshire also addressed the need for initiatives to reduce patient and site burden, such as transportation services and the use of local physicians for specific assessments. These operational considerations are crucial for successfully implementing pragmatic trials, ensuring they are both efficient and reflective of real-world clinical practice. By addressing these challenges, the industry can develop trials that are more accessible and patient-centric.

Summary

The 2025 SCOPE Summit highlighted the transformative potential of pragmatic trials in bridging the gap between clinical research and real-world practice. By focusing on regulatory support, innovative trial designs, and operational considerations, the summit underscored the importance of these trials in enhancing the relevance and applicability of clinical research. As the industry moves forward, the insights gained from this summit will be crucial in shaping the future of clinical trials, ultimately improving patient outcomes and advancing clinical research.