Pre-Conference Workshop Day
Workshop A
08:00 - 10:00
8:00 am Translating OMICS & Real-World Evidence into Meaningful Insights for Target Discovery & Clinical Trial Design
Synopsis
The integration of OMICS data and real-world evidence is transforming drug development. With the increasing volume of CKD data generated through OMICS technologies and real-world evidence studies, there is a critical need to translate this information into actionable insights.
This workshop will explore how to leverage OMICS findings to enhance our understanding of disease mechanisms and identify novel therapeutic targets. Participants will learn how to translate these insights into effective clinical trial strategies, improving patient stratification and driving better outcomes in CKD and beyond, all to equip attendees with the knowledge to navigate the complexities of data-driven drug development
This workshop will gather experts to discuss:
- Discuss the current landscape of OMICS data collection and its potential to inform the identification of novel therapeutic targets
- Explore methods to integrate OMICS data with pathophysiological understanding, enabling the identification of mechanisms driving kidney diseases and guiding the discovery of new treatment avenues
- Review how RWE can inform adaptive trial designs and patient stratification, using data from kidney precision medicine projects and successful case studies
- Analyze insights from RWE studies on the importance of early CKD detection and how this can transform patient care and improve treatment protocols
- Examine comparative studies of patient pathways across different countries, highlighting the significance of understanding patient experiences in clinical trial designs.
10:00 – 10:30 am Morning Break
Workshop B
10:30 - 12:30
10:30 am Navigating Challenges in Pediatric Nephrology to Unveil a New Development Pathway
Synopsis
Pediatric nephrology presents unique challenges in drug development and trial design. This workshop will explore the critical considerations for conducting clinical and preclinical trials in pediatric populations, focusing on ethical obligations, regulatory perspectives, and innovative approaches to trial design
This workshop will cover:
- Discuss studies of underlying disease mechanisms in pediatric nephrology and how insights into pathobiology translate into new diagnostic and therapeutic approaches to enhance understanding of disease drivers and inform treatment strategies
- Explore factors influencing trial design, including parental acceptance and concerns regarding participation, while highlighting ethical responsibilities to improve recruitment efforts
- Provide examples of successful trial designs that address pediatric-specific challenges, including dose finding and endpoint selection, applicable in both clinical and preclinical settings to equip you with practical strategies for designing effective pediatric trials
- Understand the importance of translating adult data for pediatric use and the challenges involved, including age-specific considerations in clinical and preclinical studies to facilitate the development of safe and effective treatments for children
- Review the FDA’s requirements for pediatric trials, including study protocols and conditions for data extrapolation, and their implications for clinical and preclinical trial design to ensure compliance and enhance the robustness of pediatric studies.
12:30 – 1:30 pm Lunch Break
Workshop C
1:30 - 3:30
1:30 pm Mastering Hierarchical Composite Endpoints: Challenges & Opportunities in CKD Trials
Synopsis
Hierarchical composite endpoints (HCEs) combine multiple clinical and surrogate endpoints in a single trial, providing a pathway to demonstrate efficacy across various outcomes in CKD trials. However, the complexity of developing, ranking, and interpreting HCEs presents unique challenges, especially in CKD trials where surrogate endpoints such as proteinuria or GFR slope play a critical role.
This workshop will provide an in-depth exploration of how to effectively design, analyze, and interpret HCEs in CKD, addressing both statistical and clinical concerns.
This workshop will cover:
- Understand the differences between HCEs and traditional composite endpoints and their application in CKD trials to gain a thorough understanding of HCEs and their role in CKD trials.
- Learn to rank endpoints by clinical significance, from mortality to surrogate markers like GFR slope, while considering their impact on patient outcomes to develop strategies for constructing HCEs that accurately reflect clinical outcomes and surrogate markers.
- Dive into the complexities of interpreting a statistically significant win ratio HCEs, and examine the clinical meaning behind ratios of 1.1, 1.2, or higher and address criticisms of surrogate endpoint dominance.
- Explore strategies to integrate surrogate endpoints without overshadowing clinically meaningful outcomes, avoiding pitfalls of less impactful markers to understand how to balance surrogate endpoints with clinical significance.