Tackling today’s healthcare challenges requires collaboration from every stakeholder, including patients, academic investigators, site investigators, regulatory agencies, pharmaceutical and device industry leaders, payers, technology companies, and more.
DCRI Think Tanks demonstrate what is possible when you bring smart people together to tackle the biggest challenges in clinical research. From developing innovative approaches to leveraging artificial intelligence, these meetings are about creating pathways to useful, focused discussions across the healthcare continuum and then moving consensus to action.
Our mission: Address the most critical gaps in clinical research by convening leaders across the healthcare industry to map the way forward in designing, conducting, and implementing high-quality, evidence-based research.
Upcoming Think Tanks
Pathway to Prevention: Heart Failure as a Use Case for Building the Roadmap for Regulatory Acceptance of Preventive Agents
August 12 and 13 at Duke in D.C.
Improved understanding of potentially modifiable risk factors and the development of specific therapies that can target that risk have enabled new opportunities to develop specific strategies for the prevention of highly morbid chronic cardiovascular diseases such as heart failure (HF). Despite these opportunities, the clinical and regulatory pathways to develop evidence supporting interventions for the primary prevention of HF remain poorly defined. Key challenges include the uncertainty around acceptable endpoints and evidentiary thresholds to support regulatory approval in primary prevention, identifying the correct patients who will benefit from therapies for primary prevention, and the complexity of designing trials in populations with low near-term event rates. Using primary prevention of HF as a central use case, the Pathway to Prevention Think Tank will explore strategies to generate robust, decision-grade evidence to support regulatory approval of agents intended for primary prevention.
Participants will examine critical challenges in the design and interpretation of HF prevention trials, including delineation of appropriate populations for study and use of enrichment strategies to identify patients most likely to benefit, selection of appropriate endpoints, and study design considerations (e.g, treatment duration, placebo use, safety and tolerability, and treatment crossover). The discussion will also address the operational and economic feasibility of large-scale, long-duration prevention trials, including opportunities to leverage pragmatic designs, real-world data, and platform approaches to improve efficiency.
In parallel, the group will evaluate regulatory expectations for primary prevention indications, including definitions and evidentiary standards that differentiate primary prevention from disease modification or early treatment, acceptable risk-benefit thresholds, and requirements for post-approval evidence generation.
The goal of this session is to generate actionable, consensus-driven recommendations that address the scientific, clinical, and regulatory barriers to primary prevention trials—ultimately enabling more efficient development and approval of interventions that reduce lifetime risk of heart failure and improve population-level cardiovascular health.
KEY QUESTIONS:
• How are primary prevention trials for chronic diseases such as HF similar or different from strategies around more traditional prevention trials focused on atherosclerosis?
• How can we design trials that provide robust data on the efficacy and safety of primary prevention strategies that can effectively inform decisions from clinicians, regulators, payers, and patients? What should FDA guidance include? What practical checklist is needed to support regulatory acceptance, and how can engagement with regulators and payers be better aligned and initiated earlier?
• Where are the critical data gaps, and what evidence is needed to advance primary prevention indications? How do we define the event we’re preventing and the first event/diagnosis (e.g. outpatient diagnosis of HF vs. a HF hospitalization)?
• What are appropriate surrogate and/or intermediate biomarkers/endpoints to support primary prevention? How can these surrogate or intermediate endpoints be validated and “safety-proofed” for the prevention claims and what are the expectations for post-approval monitoring?
• What are the optimal study designs for primary prevention populations (e.g., enrichment strategies, placebo use, population definition, duration, endpoints, and safety standards), and how can these trials be operationalized efficiently?
This Think Tank will be led by:
Duke University
Baylor Scott & White
Duke Research Fellow
Expert Meetings & Resulting Insights
For over 30 years, DCRI Think Tanks have convened leaders across the healthcare industry to map the way forward in designing, conducting, and implementing high-quality, evidence-based research. Below, learn about our most recent meetings and read insights from leading experts, or visit our archive of meetings and publications.
Recent DCRI Think Tanks
Patient experience data (PED) holds significant promise for informing regulatory decision-making, yet its full potential remains underutilized across the medical product lifecycle. Gaps in early planning, misaligned evidentiary expectations, and unresolved questions about stakeholder accountability continue to limit the effectiveness of PED generation and application. This DCRI Think Tank brought together regulators, sponsors, methodologists, payers, academics, and patient advocacy representatives to explore how PED can be more intentionally integrated to support benefit-risk assessments, regulatory labeling, and broader healthcare decision-making.
Meeting attendees discussed the following themes and opportunities for action:
- How should PED be integrated into clinical development plans from the outset, and what does early, iterative engagement with regulators look like in practice?
- What mixed-methods approaches (combining qualitative research, patient-reported outcomes, and patient preference information) are needed to fully capture the patient experience, and when should each be used?
- What is the appropriate evidentiary bar for PED, and how does it differ across regulatory approval, payer coverage, HTA decisions, and real-world adoption?
- How can stakeholders move from "regulatory-grade" to "decision-grade" evidence, and what does that shift require in practice?
- What are the barriers to global harmonization of PED evidentiary expectations across regulators, HTA bodies, and payers, and what would a harmonized framework look like?
- When should legacy endpoints and instruments be adapted versus replaced, and how do stakeholders balance methodological rigor with feasibility?
- How can patient burden from lengthy or redundant questionnaires be minimized without compromising data quality?
- What incentives or accountability mechanisms are needed to ensure robust PED collection is prioritized across the product lifecycle, including post-market phases?
- How can data-sharing agreements, consent language, and publication norms be structured to encourage the dissemination of PED findings, including from negative trials?
Learn More About This Think Tank
Directors: Emily O'Brien, PhD, DCRI, and Amylou Dueck, PhD, Mayo Clinic; Meeting Fellow: Tina Cheng, Duke University
From administrative efficiencies to early-stage drug discovery and trial optimization, artificial intelligence (AI) is reshaping the clinical research landscape. The field continues to explore practical applications of AI, including integration into regulatory review, as well as opportunities in late-stage clinical development and post-market surveillance. These expanded applications present new opportunities and challenges that demand thoughtful exploration.
This Think Tank brought together leading experts from across the clinical research ecosystem, including academia, industry, regulatory agencies, patient advocacy, and others, to explore the evolving role of AI in the later stages of clinical development. The program examined how AI has been supporting the execution, conduct, and analysis of the trials, including data collection, transformation, and interpretation. The program also evaluated the standards that have been applied to AI use cases and how they should be developed to inform efficient, transparent, and ethical use of AI within clinical research.
As AI capabilities accelerate, the clinical research field must address the widening gap between technological potential and practical implementation. This program explored how to responsibly advance AI adoption while ensuring safety, compliance, and scientific rigor.
Key Themes and Objectives
- What has AI accomplished in clinical development to date, and what critical gaps remain? What standards have been applied for AI adoption across the clinical research ecosystem?
- How can AI support data collection, transformation, analysis, and interpretation of Phase III and Phase IV FDA-mandated studies?
- How are regulators currently leveraging AI, and what governance, safety, and evidentiary standards are needed?
- How does the FDA’s AI guidance inform algorithm use and risk-based strategies?
- What are the implications of AI-enabled safety data management and post-market surveillance?
Directors: Michael E. Matheny, MD, MS, MPH, FACMI, FAMIA, Vanderbilt, and Sreekanth Vemulapalli, MD, DCRI; Meeting Fellow: Husam Salah, MBBS, Duke University
As the clinical trial landscape evolves, particularly in the face of increasingly complex and costly outcomes trials, there is growing urgency to identify and validate alternative endpoints that extend beyond mortality and other “hard” outcomes. These alternative endpoints, which may require shorter follow-up and smaller sample sizes, offer the potential to streamline trial design, reduce resource burdens, and accelerate access to effective therapies.
This Think Tank convened a group of stakeholders to explore the opportunities and challenges of advancing surrogate and nontraditional endpoints for regulatory-enabling clinical trials. The discussion drew on cross-disciplinary expertise, with a particular focus on functional measures, surrogate endpoints, and patient-reported outcomes as meaningful indicators of clinical benefit across therapeutic areas.
This session aimed to answer the following key questions:
- Which recent surrogate endpoints gained regulatory approval, and how did disease context affect their acceptance? How quickly did regulators, clinicians, and patients adopt these endpoints?
- In what scenarios were conventional endpoints no longer practical or sufficient to capture meaningful clinical benefit? What factors (regulatory, industry, clinical) perpetuated their continued use?
- How could we expand the use and regulatory acceptability of alternative endpoints, such as imaging, functional measures (e.g., muscle or cognitive function), and patient-centered outcomes (e.g., quality of life or symptom scores)? How did practical use compare in rare versus common diseases and pre- and post-approval?
- What did “known benefit” mean in the context of regulatory certainty? What were the expectations for endpoints that were “reasonably likely to predict clinical benefit”? Did these need to evolve with recent advances in preventative care?
- What special considerations apply when designing trials for prevention or lower-risk populations? How do we take steps towards validating endpoints that inform successful prevention and “efficacy” in lower-risk populations over a manageable timeframe?
Directors: Lesley Inker, MD, MS, Tufts, and Christopher Mosher, MD, MHS, DCRI; Meeting Fellow: Kristin Corey, MD, Duke University
Primary care is the cornerstone of a healthcare system, providing comprehensive and continuous care to patients. It plays a crucial role in managing chronic health conditions, promoting preventive care, and ensuring timely access to medical services. However, due to the volume and breadth of patient care, and limited resources, primary care providers often face significant challenges in integrating clinical research and rapidly implementing research findings into practice.
Meeting attendees discussed these challenges and identified innovative strategies to move the needle on embedding clinical research into primary care practice. Key questions they considered included:
- How is primary care evolving, and where are there opportunities to better integrate clinical research? How do primary care research settings differ from other clinical research sites?
- How can we engage and support primary care clinicians to increase their participation in clinical research? How can we leverage existing primary care research networks?
- What types of clinical trials or other clinical research are most suitable for primary care settings? How do we design trials to fit into primary care settings?
- What role can technology play in supporting primary care clinicians to participate in clinical trials?
- What are the potential use cases of trials in primary care settings that bring value to patients, primary care clinicians and their health systems, and industry partners?
Directors: Rowena Dolor, MD, MHS, DCRI; Russel Rothman, MD, MPP, Vanderbilt; Meeting Fellow: Ryan M. Kane, MD, MPH, Duke University
Recent Think Tank News & Publications
Leaders and Partners
The DCRI Think Tanks Advisory Board
Advisory board partnerships with industry leaders help the DCRI Think Tanks program address the right topics, at the right time, with the right people. Our partners provide crucial insight and connections that go beyond DCRI's clinical and operational expertise. From guidance on attendees, framing discussion topics, and contributions to resulting publications, our advisory board members are players in the lifecycle of every event.
