DCRI Think Tanks Demonstrate What Is PossibleTackling 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 cardiovascular diabetes to the Pediatric Rule to the Modern Data Monitoring Committee, these meetings are about creating pathways to useful, focused discussions across the healthcare continuum and then moving consensus to action.
Our mission—to 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.
Recent DCRI Think Tanks
January 29-30, 2020
Leveraging Artificial Intelligence and Machine Learning Methods and Approaches to Transform Clinical Trial Design, Planning, and Execution
The clinical trials landscape is rapidly evolving based upon technological and data science innovations that are enabled and facilitated by advances with Artificial Intelligence (AI) and Machine Learning (ML) techniques applied to clinical trial data and processes.
Concurrently, regulatory interest in developing a new tailored review framework for artificial intelligence-based medical devices and software algorithms intersects with the applications of AI/ML to contemporary drug discovery and drug development. As pharmaceutical companies seek to harness the potential of AI/ML for improving clinical trial design, planning, and execution and the FDA develops the organizational structure and technical expertise for appropriate oversight of AI/ML techniques utilized for health care delivery and clinical research, thoughtful consideration of the perspectives from a diverse group of stakeholders is needed to delineate the appropriate review framework and expectations. The Duke Clinical Research Institute seeks to bring academic, regulatory, and industry leaders, and AI/ML subject matter experts together to discuss key questions and topics related to the application of AI/ML techniques to enable the clinical trials of the future with appropriate quality expectations and to develop agreement on risk mitigation strategies that promote innovation and efficiency.
Key objectives of this DCRI Think Tank included:
- A review of recent experiences with AI/ML for informing and supporting clinical trials
- The delineation of data, methodological, and resource needs to refine and validate AI/ML approaches that will meet quality expectations for use with clinical trials
- A determination of the regulatory framework for oversight and approval of AI/ML approaches for supporting clinical trials especially for outcome classification
- Discussion on the use of deep learning for casual inference in observational settings for RWE
October 2-3, 2019
Innovative Approaches to Accelerating New Drug Evaluation for Chronic Cardiometabolic Diseases - Leveraging Intermediate Non-Clinical Endpoints and Pragmatic Clinical Trials
The development of new therapies for chronic cardiometabolic diseases has become increasingly expensive and time-consuming as the pivotal clinical trials required to demonstrate safety and efficacy on clinical outcomes have gotten larger and longer in duration. This reality means that many promising new therapies for chronic cardiometabolic conditions are abandoned and never developed. One often proposed solution to this challenge is to accelerate development through pre-market clinical trials with intermediate, non-clinical “surrogate” endpoints to be followed by post-market confirmatory, pragmatic clinical trials with hard clinical outcomes.
This Think Tank focused on the question of whether and under what circumstances such an approach would be feasible and desirable. The characteristics of appropriate, intermediate, non-clinical endpoints that meet the needs of all stakeholders were reviewed and discussed. Clinical trials evaluating the effects of novel therapies on non-clinical endpoints will require subsequent confirmatory clinical trials with clinical endpoints. The design and timing of these confirmatory clinical trials were discussed with a focus on opportunities for pragmatic approaches. Not all confirmatory clinical trials will succeed. What happens when confirmatory clinical trial are not timely, adequate, or do not confirm the findings of clinical trials on non-clinical endpoints were also considered.
Key questions of this DCRI Think Tank included:
- When is accelerated development using non-clinical endpoints appropriate?
- What are the characteristics of a valid, non-clinical endpoint?
- What are the characteristics of an appropriate confirmatory clinical trial?
- When should confirmatory clinical trials be started and completed relative to regulatory review and approval?
- What is the potential for more pragmatic approaches to confirmatory clinical trials?
- What happens when confirmatory clinical trials are not conducted adequately or in a timely fashion?
- What happens when confirmatory clinical trials do not confirm the results of clinical trials with non-clinical endpoints?
- What are the perspectives of all stakeholders including drug developers, regulators, investigators, clinicians, and patients?
DCRI Think Tanks Highlights
Impact of Regulatory Guidance on Evaluating Cardiovascular Risk of New Glucose-Lowering Therapies to Treat Type 2 Diabetes Mellitus: Lessons Learned and Future Directions
The authors of a recent paper published in Circulation summarized the key takeaways from a February 2018 DCRI Think Tank on finding a more flexible path for diabetes drug approval. The gathering of representatives from academia, industry, regulatory agencies, and the NIH addressed guidance issued by the FDA in 2008 that glucose-lowering therapies cannot receive approval until evidence is provided that they do not increase the risk of major adverse cardiovascular events. While envisioning a new path forward, the authors balanced safety concerns, cost, evidence generation, and trial design.
Technology-Enabled Clinical Trials
In October 2018, a diverse stakeholder group convened in Washington, DC, to examine how electronic health record, mobile, and wearable technologies could be applied to clinical trials. The group specifically examined how these technologies might streamline the execution of clinical trial components, delineated innovative trial designs facilitated by technological developments, identified barriers to implementation, and determined the optimal frameworks needed for regulatory oversight. The group concluded that the application of novel technologies to clinical trials provided enormous potential, yet these changes needed to be iterative and facilitated by continuous learning and pilot studies.
Heart Failure Preserved Ejection Fraction
The number of persons with heart failure has continued to rise over the last several years. Approximately one-half of those living with heart failure have heart failure with preserved ejection fraction, but critical unsolved questions remain across the spectrum of basic, translational, clinical, and population research in heart failure with preserved ejection fraction. In this study, the authors summarize existing knowledge, persistent controversies, and gaps in evidence with regard to the understanding of heart failure with preserved ejection fraction. The following analysis is based on an expert panel discussion “Think Tank” meeting held in June 2017, that included representatives from academia, the National Institutes of Health, the U.S. Food and Drug Administration, the Centers for Medicare & Medicaid Services, and industry.
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.