SCOPE Preview: Patient-Centricity, Real-World Evidence, and the Evolution of Clinical Trial Design

January 10, 2023 | At the 14th Annual Summit for Clinical Ops Executives—held February 6-9, 2023, in Orlando—the wealth of offerings for the clinical research community is once again astounding. With 27 different conferences, three plenary keynote sessions, two awards ceremonies, cross-department panels, the Master of Clinical Research golf tournament, and interactive breakout discussions, the programming is more than enough to keep over 2,200 leaders in clinical operations and research busy for four days. As we begin to work through the agenda, here are a few of the sessions and events that we are flagging. –The Editors 

Keynotes and Golf in Orlando 

Each year the SCOPE event is known for convening the best in both conversation and recreation and this year’s plenary program—and the second annual golf tournament—is no exception. The week features three keynote sessions. On Monday, February 6, a panel of collaborating partners including representatives from Novartis, Sanofi, Merck, Howard University Hospital, and Morehouse School of Medicine will discuss “Beacon of Hope”, a 10-year, $33.7M commitment from Novartis and the Novartis US Foundation to increase diversity among clinical trial participants and investigators; improve access to high-quality education and promising jobs; address inherent bias in the data standards, and find actionable solutions to environmental and climate issues that disproportionately affect health among communities of color.  

On Tuesday, February 7, Virginia Nido, Genentech, will challenge the audience to make real action on patient-centricity, and stop “admiring” the problem. “We need to get real,” she says. “We still have so far to go and together we must continue to drive an uncomfortable level of change.” Next, a second plenary panel with representatives from Takeda, Otsuka America, Tufts University, and Daiichi Sankyo will explore what clinical research has learned about navigating global crises, be it a pandemic, war, hyperinflation, supply chain disruptions or more.  

Finally on Wednesday, February 8, in a pair of fireside chats, Amy Abernethy, former Principal Deputy Commissioner, FDA and Christopher Boone, AbbVie, will discuss the increasing role and opportunities for real-world evidence in regulatory pathways and next-gen data sources. (In January, Boone spoke about real-world data on The Scope of Things podcast.) Then a group of pharma executives from Pfizer, Merck, and Janssen will chat about building a multi-year roadmap for future-ready operations.  

But SCOPE has always been at least as much of a networking destination as a learning opportunity. The Masters of Clinical Research—SCOPE’s annual golf tournament—returns on Monday, February 6, a perfect excuse to enjoy Orlando’s February weather.  

2023 Award Winners 

This year SCOPE plays host to two industry award programs. The first, SCOPE’s Participant Engagement Award is now in its 7th year highlighting innovation and change in how the clinical research industry communicates with participants in the fields of recruitment and retention in clinical trials. Finalists for 2023 were named last December, and representatives from 1nHealth, Cedar Health Research, ClinOne, ProofPilot, and StudyKIK will present their projects to the judging panel live during the opening keynote session on Monday, February 6.  

New this year, the SCOPE Best of Show Awards from Clinical Research News and ClinEco empowers the SCOPE attendees themselves to highlight the best new products powering clinical research. The SCOPE Best of Show Awards are open to any exhibiting company at SCOPE. Finalists will be announced before the event, and the entire SCOPE community will vote for the Best of Show winners live at the event and the 2023 SCOPE Best of Show winners will be named on Wednesday, February 8.  

Co-Located Events 

In addition to the regularly schedule SCOPE content, two additional forums will be co-located with the event this year. Barnett's Clinical Research Training Forum brings together industry training/learning and development professionals from across the globe to share approaches to innovative training program design and team development. During this 1.5-day event, speakers will share case studies and their approaches to developing training programs that encourage critical thinking skills and the development of organizational talent while fostering employee retention.  

The inaugural SCOPE Venture, Innovation, & Partnering Conference launches this year, aiming to bring together C-level executives from investment firms that support innovation in the clinical trial process, strategics from pharmaceutical and leading CROs, both venture and corporate arms who have a vested interested in driving this innovation, and also CEOs and innovators from industry, driving the technology in this space.  

Learn, Connect, Innovate 

The heart and soul of the event, though, remains the presentations from researchers, site staff, vendors, study participants, and others who are intimately connected with clinical research, its goals, and its outcomes. The planned talks are broad and rich, sharing in depth looks at what tools, technologies, and approaches are bearing fruit in clinical research.  

Updates from DTRA 

Craig Lipset, Co-Chair, Decentralized Trials & Research Alliance (DTRA), gives an update on DTRA’s mission to enable collaboration of stakeholders to accelerate the adoption of patient-focused, decentralized clinical trials and research within life sciences and healthcare through education and research. (Lipset recently outlined our decentralized future for The Scope of Things podcast.)  

Accelerating Clinical Trial Feasibility with Data Science 

Corey Jones, Johnson & Johnson, will explore how the clinical trial feasibility process can be enhanced with data science and technology. Jones will share the journey of developing applications and insights to move an organization relying on Excel and other offline, fragmented solutions to a connected, efficient solution generating real-time insights for better site selection. 

Predictive Modeling for Feasibility and Patient Enrollment 

Accurate forecast of a clinical trial enrollment timeline at the planning stage is of great importance to both corporate strategic planning and trial operational excellence. Li Wang, AbbVie, proposes a new statistical framework based on generalized linear mixed-effects models (GLMM) and non-homogeneous Poisson processes to model the country initiation, site activation, and subject enrollment sequentially in a systematic fashion. Substantial improvement in prediction accuracy is observed when applied to 30 real studies. 

Retention Strategies Centered around Participants and Results 

The ongoing Parkinson’s Progression Markers Initiative study enrolled more than 1,000 participants between 2010 and 2018 at 33 sites in 10 countries in a longitudinal, observational study involving repeat biological sampling, imaging scans and clinical assessments. Maggie Kuhl, Michael J Fox Foundation for Parkinson’s Research, highlights a primary focus of the Foundation’s retention strategy: to involve and interact with participants, which aligns with the field’s current emphasis on patient-focused drug development. Kuhl will share pillars of retention/engagement strategy and examples of varied approaches (e.g., in-person events, “share your story” forms, newsletters, conference calls, participant profiles) to consider in building their own study retention strategies. 

Optimizing Clinical Trial Agreements and Budgets  

Managing clinical trials, of any size and complexity, requires strategic planning, negotiations of agreements/budgets to ensure adequate resource allocation, risk mitigation and cost-efficient execution. Marina Malikova, Boston University School of Medicine, shares how trial management regulatory guidelines can be standardized further, and robust methods of evaluation of study protocol complexity developed in conjunction with risk management strategies, adequate provisions in clinical trials agreements and accurate budgeting are needed 

Outsourcing to Technology Vendors: Contracts, Metrics, and Challenges 

With decentralized clinical trials and patient centricity being accelerated in the light of the COVID-19 pandemic, there are a plethora of suppliers offering wearable, biosensors, devices, and app services. Nick Lewis, Bayer, discusses how to ensure the technology vendor solutions are fit for purpose, the supplier can meet sponsor expectations, and the clinical trial proceeds according to plan.  

Best Practices for Assessing CRO Performance 

With many companies using some form of CRO outsourcing model, including FSP arrangements, it is increasingly important to have effective performance measures that help assess the overall relationship and performance not only at an individual study level but also at a portfolio level. A panel of executives from Daiichi Sankyo, ICON, Merck, Jazz Pharmaceuticals, and Dorricott Metrics & Process Improvement will discuss what approaches have worked well, and what haven’t worked well including incentive models (bonus, earn-backs, etc.). 

Automating Clinical Database Build 

Ralph Russo, Pfizer, will showcase methods to minimize the cycle time for database build and highlight a simple method to leverage standard metadata to iteratively prototype the clinical database while the protocol matures. He plans to explore emerging methods that build upon this concept to further reduce cycle times and increase quality.  

Natural Language Generation in Clinical Research  

Natural language processing is a conventional approach, says Brian Martin, AbbVie, and he argues that natural language generation as the next step. Martin says it will be a game-changing solution once it moves to the mainstream.  

Graph Machine Learning Meets Clinical Ops 

Shameer Khader, AstraZeneca, shares the lessons learned and best practices from building TrialGraph, a technology solution to intelligently optimize tasks, including side effect prediction.  

Traditional analytical approaches cannot handle variables' sparsity or high-dimensional relationships, Khader says. He plans to share his own experiences building enterprise-scale analytic solutions to accelerate clinical development.  

What to build an AI model? Start with a data strategy first. 

Accelerating speed of delivering medicines to the people who need them starts with understanding the end-to-end data flow, says Victoria A. Gamerman, Boehringer Ingelheim. This includes organizational models that have capabilities to describe data with its biases and leverage metadata to support the speed of finding necessary information contained in the data. The solution starts with a holistic Data Strategy that lives at the intersection of Clinical Strategy and Digital Strategy. (Hear more from Gamerman on the Trends from the Trenches podcast where she discusses real-world data in drug discovery.)  

The Transforming Landscape of Clinical Trial Locations  

The traditional dedicated clinical trial site approach model has served its purpose for years, but it's time for the industry to embrace more evolved approaches to clinical trial locations. Speakers from CVS Health, Walgreens, ZS, and Walgreens Boots Alliance explore new locations, optimizing protocols for decentralization, and maintaining patient engagement.  

DCT by Design  

Jade Dennis and Sara Doshi, both of Eli Lilly, explore how to use a Design Studio to optimize the schedule of activities (SoA) for decentralized clinical trials. A web-based design studio, backed by an SoA taxonomy, enables exploration of SoA designs that streamline trial delivery and support decentralization opportunities, they say. As the digital design library grows and more studies deploy DCT capabilities, frequency of DCT use and impact to downstream operational metrics will be critical to future study success 

Causal Inference and Real-World Evidence Generation 

With the growing interest in augmenting traditional randomized trials with data from other sources, there has been tremendous progress in the methodological and regulatory areas. Demissie Alemayehu, Pfizer, provides a review of emerging themes in the literature and address strengths and weaknesses of the trending approaches. Special emphasis will be given to current approaches around the issue of bias control, robustness of inferential validity, and use of modern analytics. 

Registries for RWE: Challenges and Opportunities 

Rachel E. Sobel, Regeneron Pharmaceuticals, will review the recent RWE guidance from FDA and EMA on registries, and describe some of the challenges and opportunities in using registries to support safety and effectiveness evaluations, with a focus on rare disease, pediatric, and pregnancy registries, including some practical examples.  

Pragmatic Trials 

Pragmatic trial designs can build the bridge between clinical trials and clinical practice, providing valuable real-world evidence, with the rigor of randomized clinical trials, to inform treatment decisions of payers, clinicians, employers, patients, and for regulatory purposes moving forward. Helene Nordahl, Novo Nordisk, discusses her experience leveraging real-world healthcare data and linkage to claims data to address novel endpoints. She will share operational lessons learned from conducting pragmatic trials along with case examples 

Human Tissue Policies  

As the industry hurtles down the path of digital transformation, the bulk of discussion has been on new tools and technologies and the interesting ways that the data produced from them can be analyzed, with occasional discussion of the need to develop data governance models. Yet there has been relatively no discussion about the development of human tissue policies and governance models says Brenda Yanak, Clinical Transformation Partners. Yanak’s presentation will examine the critical need for equivalent focus on development of policies governing the use of human tissue and recommended components of such a policy, to ensure that organizations position themselves for success 

How the Patient Voice Shaped Sampling Strategy 

Over the past decade, Merck has conducted numerous trials involving patient-centric sampling, an enabling technology for decentralized trials. Melanie Anderson and Jennifer Campbell, both of Merck, outline their findings from patient preference questionnaires included in multiple trials. Patients prefer at-home sampling methods with novel collection devices, and preference is dependent upon sampling frequency, sampling methods, pain tolerance, and physiological condition. Data indicate that effective patient-centric sampling approaches should be painless, simple for users to execute, and minimize sample volume. This participant feedback has shaped Merck’s patient-centric sampling strategy and has enabled us to implement sampling approaches that are truly patient-centric 

Gamifying Clinical Trials for Kids  

Kids are not small adults and developing a medical device or a mobile health app for children requires not only originality and creativity, but also extensive knowledge of developmental psychology and child behavior. Amir Lahav, Mitsubishi Tanabe Pharma America, believes it is particularly important to create age-appropriate medical content and attractive graphic illustrations in an effort to keep children engaged throughout the clinical trial journey. Gamification is a key element for implementing incentivized strategies combined with accessible technology to ensure protocol adherence and retention of trial participants. The child's mental well-being is often not receiving enough attention at the protocol design stage.  Lahav will outline areas where we can do more to improve the clinical trial experience, making it more friendly for both parents and children This may include the use of video animation in eConsent materials; virtual awards based on the child's preferences; gamed-based clinical trial software platform; and augmented reality to alleviate stress and anxiety before complex medical exams such as MRI, muscle biopsy, and phlebotomy.