Episode 8: Are We Any Closer to Patient-Centric Trials?

This video provides an in-depth exploration of the current state of patient-centric clinical trials, viewed through the lens of a mid-sized Contract Research Organization (CRO). Host Richard Young, VP of Strategy at Veeva Vault CDMS, speaks with Alan Morgan, CEO Advisor at Excelya, about the persistent challenges and emerging opportunities in making trials more patient-focused. The discussion highlights the disconnect between significant industry investment in "big data" tools for site selection and the actual improvements in patient recruitment, emphasizing that traditional methods and strong site relationships remain crucial.

Alan Morgan details Excelya's strategy as a mid-sized CRO, focusing on quality, speed, and a "centers of excellence" approach. These centers include specialized services in statistics and programming, data management, pharmacovigilance, electronic Trial Master File (eTMF) – notably in partnership with Veeva – and medical writing. He stresses the importance of technology as an enabler for cost and speed, while acknowledging that smaller CROs often need to partner with larger technology providers rather than developing proprietary solutions. The conversation also delves into the critical role of patient advocacy groups in achieving a truly diverse and global patient voice, especially given the varying standards of care across different regions.

A significant portion of the discussion centers on the evolution of data management and the persistent issues that hinder clinical trial efficiency. Morgan laments the lack of dramatic improvement in patient accrual metrics over 25 years, despite extensive investment in data mining and big data for site identification. He argues that the role of the data manager has transformed from a "poor relation" to a dynamic, proactive integrator and custodian of complex, multi-source data. The speakers also identify protocol changes as a major source of cost, waste, and inefficiency in the drug development process, advocating for more rigorous upfront planning and higher hurdles for amendments. The episode concludes with a call for greater industry collaboration and interoperability, referencing initiatives like TransCelerate, to collectively advance clinical research for the benefit of patients.

Key Takeaways:

• Mid-sized CROs have a distinct market opportunity: Excelya's strategy emphasizes quality, speed, and specialized "centers of excellence" in areas like data management, pharmacovigilance, and eTMF, catering to clients seeking focused expertise.
• Technology partnerships are crucial for CROs: Smaller CROs cannot sustain the investment for state-of-the-art proprietary technologies and must partner with larger providers (e.g., Veeva for eTMF) to leverage advanced solutions and drive efficiency.
• Big data's impact on site selection and patient recruitment is underwhelming: Despite significant investment in "big data" tools for site identification, overall patient accrual metrics have not dramatically improved over the last 25 years, indicating a gap between technology promise and real-world results.
• Traditional site relationships remain vital: Professional sites and established relationships, often requiring in-person engagement, are still fundamental for effective patient recruitment, highlighting the limitations of purely data-driven approaches.
• Patient advocacy groups are essential for patient-centricity: To truly incorporate a diverse global patient voice into trial protocols and address varying standards of care, strong, professional, and sustainable connections with patient advocacy groups are indispensable.
• The role of data management has significantly evolved: Data managers are no longer merely transactional but have become critical integrators and custodians of increasingly complex, multi-source data, requiring smarter management and critical thinking skills.
• Offshore data management teams are becoming client-facing: There's a dramatic shift in offshore teams (e.g., in India) from simple task execution to direct client engagement, managing data and interacting with US and Western European customers.
• Protocol changes are a major industry inefficiency: Frequent protocol amendments are costly, wasteful, and risk compromising trial integrity. The industry needs to prioritize getting protocols right upfront and introduce higher hurdles for changes.
• Critical thinking is a vital skill across clinical trial teams: The industry needs to invest more in training and developing team members (from CRAs to data managers) to foster critical thinking, beyond mere SOP compliance, to navigate complex processes.
• Data integration complexity hinders FDA approvals: The proliferation of diverse data sources (e.g., e-pro, mobile data) makes data integration and database lock challenging, contributing to basic errors and missing data that can lead to FDA non-approvals on first submission.
• Industry collaboration and interoperability are key to progress: Organizations like TransCelerate are crucial for driving coordinated action and addressing interoperability challenges across the industry, particularly in data-focused activities.
• The line between competitors and partners is blurring: In the complex landscape of clinical trials, organizations often find themselves partnering on one project and competing on another, necessitating strong trust and quality alignment.

Tools/Resources Mentioned:

• Veeva (Vault CDMS, eTMF platform): A key technology partner for Excelya, particularly for electronic Trial Master File solutions.
• Excelya: A mid-sized full-service CRO based in Europe and India, with emerging US presence.
• TransCelerate: An industry initiative focused on improving clinical trial efficiency and interoperability.
• Panthera: An example of a Site Management Organization (SMO) in the UK.
• Cystic Fibrosis Foundation: Cited as a highly professional patient advocacy group with its own IP and domain knowledge.

Key Concepts:

• Patient-Centric Trials: The core theme, discussing efforts and challenges in designing and executing clinical trials with the patient's perspective and needs at the forefront.
• Site-less Trials/Hybrid Models: Discussions around decentralized trial approaches, noting that while there's investment, the market hasn't fully shifted away from traditional site-based recruitment.
• Centers of Excellence: A strategic approach by Excelya to build standalone strength and expertise in specific non-clinical monitoring and project management activities (e.g., data management, eTMF, pharmacovigilance).
• Electronic Trial Master File (eTMF): A digital system for managing essential clinical trial documents, highlighted as a key area of focus and partnership with Veeva.
• Protocol Amendments: Changes made to a clinical trial protocol after it has been initiated, identified as a significant source of cost, delay, and operational complexity.
• Data Management Evolution: The shift in the data manager's role from a low-status, transactional position to a high-value, proactive, client-facing role as an integrator and custodian of diverse data sources.