Aliza Thompson
Director of the Division of Cardiology and Nephrology, Center for Drug Evaluation FDA
Seminars
As therapeutic innovation expands across both rare glomerular diseases and more common causes of CKD, developers continue to face complex questions around endpoint selection, evidence expectations, and patient relevance. This workshop brings together regulatory, clinical, and industry perspectives to explore how surrogate endpoints, patient‑reported outcomes, and real‑world evidence can be thoughtfully applied to strengthen trial design.
Across two expert presentations and an interactive fireside chat, faculty will discuss learnings from C3G and other rare disease programs, considerations for trial design in more common CKD conditions, and evolving expectations around background therapies and standard of care. Participants will then work through facilitated discussion groups on post‑marketing evidence, treatment guidelines, patient identification, and payer‑relevant business cases to translate these insights into practical development strategies.
This session is designed to help teams advance the science of CKD drug development by aligning earlier with regulators, refining endpoint selection, and incorporating patient‑centered approaches that meaningfully strengthen the evidentiary package.
Key Objectives:
- Evolving use and interpretation of surrogate and composite in rare and common kidney diseases
- Approaches to trial design in therapeutic landscapes with multiple background therapies and established guidelines
- Integrating patient‑reported outcomes, disease‑specific function measures, and real‑world evidence to enhance trial relevance and reduce patient burden
- Lessons from recent development programs in C3G and other rare kidney conditions, focused on regulatory science, collaboration, and endpoint development
- Practical considerations for post‑marketing evidence, payer expectations, treatment guideline positioning, and improving diagnosis and access for diverse CKD populations
- The new Kidney HCE framework combines GFR slope, various thresholds of GFR decline (e.g. ≥ 40%, 50%, 57%), ESKD, and mortality in a hierarchical ordering to improve trial sensitivity and clinical relevance
- Regulatory bodies (NKF/FDA/EMA) are increasingly recognizing eGFR slope + albuminuria as valid surrogates, but the choice of thresholds, population, and follow-up duration remain critical
- Key methodological challenges include deciding component priorities, handling multiplicity, ensuring interpretability for patients
- Use-cases in broad CKD and rare subpopulations (e.g. IgA nephropathy) differ: design of HCEs must adapt thresholds, endpoints composition, and regulatory acceptability depending on disease, severity, and existing therapies
- Explore our evolving understanding of the pathophysiology linking cardiovascular, kidney and metabolic disorders
- Identify when and how overlapping CKM phenotypes can be leveraged to design more efficient clinical trials
- Discuss next steps in integrated drug development and regulatory frameworks for CKM components with shared mechanistic drivers
