Why We Need to Expand Patient Choice in Clinical Trials

This video provides an in-depth exploration of the evolution and future of clinical trials, emphasizing the critical need to expand patient choice and streamline site operations through technology. Featuring Tim Davis, VP of Strategy for MyVeeva for Patients, and Richard Young, the discussion aims to cut through the industry buzzwords like "decentralized clinical trials," "patient centricity," and "site centricity" to focus on practical, impactful strategies. The conversation traces the journey from archaic paper-based data collection to the promise of integrated digital platforms, highlighting the challenges and opportunities in managing clinical data, engaging patients, and navigating regulatory landscapes.

The speakers delve into their early experiences in clinical data management, recalling the days of red and green pens, paper CRFs, and the initial skepticism surrounding electronic patient-reported outcomes (e-PRO) on devices like Palm Pilots. This historical context sets the stage for understanding the persistent challenges in data integration, where e-PRO data was often an "afterthought" and disconnected from the main clinical data management systems. Tim Davis argues that while regulators have become more supportive of digital solutions and patient-owned devices, the industry's own hesitancy and legacy practices often hinder innovation. He redefines patient centricity as simply "choice" and site centricity as "convenience," advocating for flexible, mixed-model approaches to trials rather than rigid adherence to the "decentralized" label.

The discussion progresses to envisioning the future of "digital trials," emphasizing the necessity of a consistent, underlying platform that can flex to accommodate diverse patient needs and participation models, regardless of location. The COVID-19 pandemic is cited as a catalyst that exposed the shortcomings of disparate technology "islands" but also demonstrated the industry's capacity for rapid digital adoption. The podcast concludes with a "magic wand" thought experiment, where Tim Davis expresses a desire to overcome the historical baggage of e-PRO/e-COA, enhance patient recognition and transparency by sharing study outcomes, and eliminate the costly and often unnecessary practice of provisioning devices to every patient. The overarching message is to embrace the learnings from recent years and bravely move forward with integrated, patient-focused technological solutions.

Key Takeaways:

• Evolution of Clinical Data Management: Early clinical trials were heavily reliant on manual, paper-based data collection, leading to significant delays (often 8-14 weeks) between data recording and its availability for analysis, hindering timely insights.
• E-PRO Data as an Afterthought: Historically, e-PRO/e-COA data was frequently treated as secondary to EDC data, often falling outside the primary purview of clinical data management teams, leading to integration challenges and being perceived as an additional burden.
• Bridging Real-time Data Gaps: While early electronic methods (e.g., pain scales on rudimentary touchscreens) offered real-time patient data, the critical challenge lay in seamlessly integrating this data into the broader study database for comprehensive analysis and regulatory submission.
• Regulators are More Supportive: Regulators are generally open to and supportive of digital solutions and the use of patient-owned devices in clinical trials, provided fundamental requirements like audit trails and data security are met. Industry hesitancy often stems from internal regulatory interpretations and a reluctance to be "first."
• Patient Centricity is Choice: True patient centricity should be understood as providing patients with genuine "choice" in their participation, including flexibility in how and where they engage (e.g., remote visits, local pharmacies) and access to digestible educational information about their disease and trial.
• Site Centricity is Convenience: Site centricity focuses on offering convenience to clinical sites through integrated technology solutions that operate "under one roof" with a single login, intuitive interfaces, and reduced administrative burden, especially when balancing multiple stakeholder demands.
• Rethinking "Decentralized Clinical Trials": The term "decentralized clinical trials" is viewed as potentially restrictive, advocating instead for "digital trials" that offer inherent flexibility—a mix of remote, in-person, and hybrid approaches—tailored to patient needs and study design, rather than a rigid, all-or-nothing model.
• Integrated Data Strategy: Data managers should adopt an "end-to-beginning" approach, considering how e-PRO data will integrate into the final data warehouse and statistical tables from the initial design phase. Consistent patient identification across all systems (EDC, RTSM, e-PRO) is paramount to avoid transcription errors and ensure data integrity.
• Overcoming E-PRO Legacy: The industry must move past outdated e-PRO/e-COA practices, such as the historical necessity of providing every patient with a company-issued device (e.g., Palm Pilots), which is now an expensive and often unnecessary barrier to innovation.
• Enhancing Patient Recognition: A significant improvement would be to "switch on" greater patient recognition and transparency by providing participants with high-level summaries of study outcomes, information on drug publication, and ongoing engagement, fostering trust and encouraging future participation.
• Eliminate Mandatory Device Provisioning: The practice of mandatorily provisioning devices to all patients is costly, logistically complex, often disliked by sites, and frequently unnecessary, as most patients possess their own, often superior, mobile devices. A choice-based model is preferred.
• Leveraging COVID-19 Learnings: The pandemic demonstrated the industry's ability to rapidly adopt digital technologies and maintain trial continuity under extreme pressure. These learnings about technology's potential and the need for adaptable operating models should inform future trial design and not be abandoned.
• Platform-Based Approach: Moving away from disparate "islands" of technology vendors towards a unified, integrated platform approach (like Veeva's ecosystem) can significantly reduce timelines, improve data flow, and streamline the management of diverse data sources.

Tools/Resources Mentioned:

• Palm Pilots: Mentioned as an early, rudimentary device used for e-PRO data collection.
• EDC (Electronic Data Capture): A standard system for collecting clinical trial data.
• e-PRO (Electronic Patient-Reported Outcomes): Electronic methods for patients to report their own health status.
• e-COA (Electronic Clinical Outcome Assessment): A broader term encompassing e-PRO and other electronic assessments.
• MyVeeva for Patients: A patient-facing technology platform from Veeva Systems.

Key Concepts:

• Patient Centricity: Redefined as providing patients with "choice" and options in their clinical trial participation, focusing on convenience and accessibility.
• Site Centricity: Defined as providing "convenience" to clinical sites through integrated and intuitive technology solutions that simplify workflows and reduce burden.
• Digital Trials: A preferred term over "decentralized clinical trials," emphasizing the use of technology to enable flexible, mixed-model patient participation (remote, in-person, hybrid) based on individual needs and study requirements.
• Data Islands: Refers to the common problem of disparate, unintegrated technology systems used across different aspects of clinical trials, leading to inefficiencies and data management challenges.

Examples/Case Studies:

• Early Osteoarthritis Studies: Use of rudimentary touchscreen devices in the early 2000s to collect pain scales from osteoarthritis patients, highlighting early efforts in e-PRO.
• Photocopied CRF Pages: An anecdote about monitors photocopying paper CRF pages for visual analog scales, which resulted in incorrect measurements (e.g., 9.7cm instead of 10cm), underscoring the critical importance of data integrity and the flaws of paper-based methods for primary endpoints.
• COVID-19 Pandemic Impact: The pandemic forced clinical trials to rapidly adopt layered technologies and adapt operating models, exposing both the challenges of disparate systems and the potential for technology to maintain trial continuity and accelerate drug development.