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.
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 will focus 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 will be 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 will be 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 will be considered.
Key questions of this DCRI Think Tank include:
- 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?
This Think Tank will focus on the question of how to sustain research-supported interventions at local health care institutions but also widely apply them beyond the initial systems under study. Healthcare environments around the world are increasingly driven by resource constraints and focused on maximizing health care value. Thus, descriptive studies of barrier/facilitators of implementation success are critical, but these need to link to rigorously controlled studies that evaluate the effectiveness of practice-based improvement initiatives. Implementation study outcomes impact both practice and the ability of interventions to be scaled.
Key questions of this DCRI Think Tank include:
- What are best practices in the design of implementation science studies?
- What do implementation science studies contribute to lifecycle management of a drug or device?
- What kind of real world evidence (RWE) do these implementation studies generate?
- How can complex health interventions be assembled to change clinician and patient behavior?
- How do you get patients involved and engaged meaningfully?
- How do you describe and adapt interventions to different health care settings?
- How can we best understand implementation science from a consumer perspective to help stakeholders interpret intervention benefit/value?
Cardiovascular Outcomes and Diabetes Drugs: Lessons Learned and Path Forward
DCRI faculty joined a DCRI Research Forum panel on March 6, 2018 to share their perspectives on Cardiovascular Outcomes and Diabetes Drugs after attending a think tank meeting on the same topic.