Episode 10: Why We Need to Expand Patient Choice in Clinical Trials

This video provides an in-depth exploration of the evolution of clinical trials, focusing on data management, patient and site centricity, and the future of digital solutions. Richard Young interviews Tim Davis, Vice President of Strategy for MyVeeva for Patients, who offers a historical perspective on clinical data management, from the early days of paper Case Report Forms (CRFs) to the advent of electronic data capture (EDC) and electronic patient-reported outcomes (e-PRO). Davis highlights the initial challenges of integrating e-PRO data, which was often treated as an afterthought or "another source of pain" by data managers, primarily due to its non-traditional nature compared to typical EDC source data. The discussion underscores the significant lag (often 8-14 weeks) between data collection and its visualization or integration in paper-based systems, contrasting it with the real-time insights offered by electronic methods.

The conversation delves into the role of regulators, with Davis asserting that regulatory bodies have been largely supportive of technological advancements, particularly regarding the use of patients' own devices, provided fundamental requirements like audit trails and data security are met. He notes that the industry's hesitancy, rather than regulatory barriers, often impedes innovation. A significant portion of the discussion critiques buzzwords like "patient centricity," "site centricity," and "decentralized clinical trials" (DCTs). Davis redefines "patient centricity" as offering "choice" and convenience, rather than aiming to "delight" patients who are often struggling with illness. For sites, centricity means providing convenience through integrated, intuitive solutions under a single login. He expresses a strong dislike for the term "decentralized clinical trials," arguing that many of its components have existed for decades and that the term itself has become a barrier to a clear path forward.

The speakers reflect on the impact of the COVID-19 pandemic, which forced a rapid adoption of digital tools but often resulted in a fragmented "layered tech" approach, overwhelming sites and patients with disparate systems. This experience, while challenging, underscored the need for scalable, repeatable models for digital trials. Davis envisions the future of digital trials as being driven by flexible, consistent underlying platforms that can adapt to various operating models—whether remote, in-person, or a hybrid—to truly offer patient choice. He emphasizes focusing on "how" patients participate rather than "where." The interview concludes with practical advice for data managers, urging them to "think about the end at the beginning" by involving themselves in the design phase of e-PRO solutions and ensuring consistent patient identifiers across all systems to avoid downstream issues. Davis also shares his "magic wand" wishes: to shed the historical baggage of e-PRO/eCOA development, enhance patient recognition and transparency by sharing study outcomes, and eliminate the costly and often unnecessary practice of provisioning devices to every patient.

Key Takeaways:

• Evolution of Data Management: Clinical data management has progressed from manual, paper-based systems (CRFs, validated rulers for pain scales) with significant data lag (8-14 weeks) to electronic data capture (EDC) and e-PRO, offering real-time insights.
• e-PRO Integration Challenges: Historically, e-PRO data was often an afterthought, not fully integrated into clinical data management processes, and seen as a separate "source of pain" due to its non-traditional, non-queryable nature compared to typical EDC data.
• Regulator Support vs. Industry Hesitancy: Regulators (e.g., FDA) are generally supportive of new technologies like patient-owned devices for data collection, provided core requirements like audit trails and data security are met. The primary barrier to innovation is often internal industry hesitancy and a reluctance to be "first."
• Redefining Patient Centricity: True patient centricity is about offering "choice" and convenience, not "delight." This includes providing flexible participation options (remote, in-person, hybrid), accessible educational information, and timely support tailored to a patient's journey.
• Site Centricity for Efficiency: Site centricity involves providing convenience through integrated technology solutions (e.g., single username/password, intuitive apps) that reduce burden and offer tangible benefits back to the sites, acknowledging their critical role as the "window to patients."
• Critique of "Decentralized Clinical Trials" (DCT): The term "decentralized clinical trials" is often overused and can hinder progress. Many elements of DCTs, such as patient diaries on devices, have existed for decades. The focus should be on enabling flexible participation models rather than adhering to a rigid definition of "decentralized."
• COVID-19's Impact on Tech Adoption: The pandemic accelerated the adoption of digital tools in clinical trials, but often led to a fragmented approach with "layered tech" and disparate vendor systems, creating stress for sites and patients. This highlighted the need for scalable and repeatable digital trial models.
• Future of Digital Trials: Digital trials require flexible, consistent, underlying platforms that can support a mix of remote and in-person activities, adapting to patient needs and locations. The emphasis should be on "how" patients wish to participate (e.g., day-by-day choice) rather than prescriptive "where" decisions.
• Proactive Data Management: Data managers should be involved from the very beginning of e-PRO design, considering the end-state data tables and how e-PRO data will fit into the overall data asset. This includes planning for data frequency and integration.
• Importance of Consistent Identifiers: Ensuring consistent patient identifiers (screening ID, randomized ID) across all clinical trial systems (RTSM, EDC, e-PRO) is crucial to avoid significant data management headaches and improve data integrity.
• Overcoming Historical Baggage: The industry needs to move past historical limitations and assumptions in e-PRO/eCOA (e.g., the necessity of providing specific, validated devices to all patients) to foster true innovation.
• Enhancing Patient Recognition and Transparency: Improve patient engagement and participation rates by being more transparent: sharing study outcomes, providing high-level summaries of results, and informing patients if a drug they participated in gets approved.
• Eliminating Universal Device Provisioning: The practice of provisioning a device to every patient, regardless of need, is expensive, disliked by sites, and often unnecessary, as many patients already possess better personal devices. This practice should be largely phased out in favor of patient choice.

Key Concepts:

• e-PRO (Electronic Patient-Reported Outcomes): Data reported directly by patients about their health status, symptoms, or treatment effects, collected electronically.
• eCOA (Electronic Clinical Outcome Assessment): A broader term encompassing e-PRO, e-ClinRO (clinician-reported outcomes), e-ObsRO (observer-reported outcomes), and e-PerfO (performance outcomes), all collected electronically.
• EDC (Electronic Data Capture): Software systems used to collect clinical trial data in electronic format, replacing paper CRFs.
• Patient Centricity: An approach to clinical trial design and execution that prioritizes the needs, preferences, and experiences of patients, often by offering choice and convenience.
• Site Centricity: An approach that focuses on making clinical trials easier and more efficient for investigative sites, recognizing their vital role in patient recruitment and data collection.
• Decentralized Clinical Trials (DCTs) / Digital Trials: Clinical trials that incorporate digital technologies and remote methodologies to reduce the need for in-person site visits, offering flexibility in how and where patients participate. The video advocates for "digital trials" or "distributed" over "decentralized."
• Veeva CRM: A leading cloud-based customer relationship management platform specifically designed for the pharmaceutical and life sciences industries, used for commercial operations and engagement. The speaker's role at MyVeeva for Patients indicates a focus on patient-facing technologies within the Veeva ecosystem.