Preparing for Kidney Precision Medicine Clinical Trials Workshop

Event Details

Purpose

To gain a better understanding of the kidney patient therapeutic journey and to define candidate indicators for precision medicine pathway, target and trial readiness. This workshop will identify kidney precision medicine clinical trial barrier and potential solutions and to identify concepts relative to advancing precision medicine clinical trials not yet considered.

Meeting Objectives

1. Bring in the patient's voice to advance treatments for kidney disease.
2. Define candidate indicators for precision medicine pathways, targets, and trial readiness.
3. Identify kidney precision medicine clinical trial barriers and potential solutions.
4. Identify concepts relative to advancing precision medicine clinical trials not yet considered.

Background

Progressive kidney disease affects how individuals feel, function, and survive. Therapies for individuals with kidney disease have only modest efficacy; both risk for severe toxicity and selection of treatments are not well informed by precise assessment of disease mechanism(s) active at the time of decision-making. In this standard of care context, many people with chronic kidney disease experience a series of therapies with limited efficacy and, ultimately, progress to kidney failure. Scientific advances are increasing awareness and improving the ability to assess molecular mechanisms of kidney disease and elucidation of associated therapeutic targets.

Organizing Committee

External Members

NIDDK

Registration Deadline

March 17, 2024

Agenda

Draft Agenda

March 18, 2024

9:00 a.m. – 9:20 a.m.

Welcome and Workshop Objectives

9:20 a.m. – 9:45 a.m.

Kidney Patient Treatment Challenges
Three patient speakers—TBD

Panel Q&A

9:45 a.m. – 10:30 a.m.

Precision Medicine Trial Exemplars

• Example 1 (9:50 a.m. – 10:10 a.m.)
• Example 2 (10:10 a.m. – 10:30 a.m.)

10:30 a.m. – 10:45 a.m.

Q&A

10:45 a.m. – 11:00 a.m.

Break

11:00 a.m. – 12:30 p.m.

Kidney Precision Medicine Clinical Trial Readiness Assessment

• Laboratory perspectives on biomarker discovery and validation for disease pathway identification and target engagement assessment
• Pathways, targets, and matching
• Bench-to-trial example

Q&A

12:30 p.m. – 1:30 p.m.

Lunch

1:30 p.m. – 3:00 p.m.

Breakout Group Introductions and Directions

• Science—Identify candidate indicators for actionable biomarkers, targets, and interventions. Explore mechanisms to conduct the assessment, including expertise, frequency, and venue.
• Systems—Explore infrastructure characteristics and the components required to conduct time-sensitive mechanistic assessments on an individual level to inform precision medicine trial stratification and participation.
• People: Patient, Caregiver, and Clinician Perspectives—Discuss approaches to kidney precision medicine trial goals, uncertainty, and communication. What information is needed to prepare the community for precision medicine trials for patients, clinicians, systems, and payors? How should the uncertainty inherent in clinical trials be communicated with individuals with kidney disease (e.g., co-development, consent, communication)?

Breakout Reports

3:00 p.m. – 3:20 p.m.

Break

3:20 p.m. – 3:50 p.m.

U.S. Food and Drug Administration Perspective on Precision Medicine Clinical Trials

3:50 p.m. – 4:30 p.m.

Industry Perspective on Precision Medicine Clinical Trials

4:30 p.m. – 4:45 p.m.

Wrap-up and Directions for Day 2

March 19, 2024

9:00 a.m. – 9:15 a.m.

Welcome and Plan for the Day

9:15 a.m. – 10:30 a.m.

Advancing Health through Precision Medicine
Each speaker will include strategy, health outcomes, challenges, and a solutions Q&A

• Right patients, right drug(s)
• Right dose (pharmacogenetics)
• Right time

10:30 a.m. – 10:45 a.m.

Break

10:45 a.m. – 12:00 p.m.

Breakouts on Identifying Potential Challenges to and Prevention/Mitigation Strategies for Kidney Precision Medicine Trials

1. Right Time: How do we account for compensatory mechanisms in the development of precision medicine studies? Consider the trial of a single agent versus a combination of agents.
2. Real-time Matching: What is our capacity to generate, manage, and conduct integrated analyses of high-quality disease mechanism profiling data for trial matching on a national scale?
3. Right Patients: Consider a highly specific disease mechanism in a single individual or subgroup. What are reasonable considerations and approaches to trial design and the subsequent extension of the findings to others?

For all breakouts: What are the likely obstacles and potential mitigation strategies to overcome these obstacles?

12:00 p.m. – 1:00 p.m.

Lunch and Networking

1:00 p.m. – 2:30 p.m.

Breakout Reports with Q&A

2:30 p.m. – 3:00 p.m.

Summary and Wrap-up