Abstract

Emerging Innovative Drug Development Strategies for Kidney Diseases and Beyond

Author(s): Julie Lin, Julie Lin*

Reaching end-stage kidney failure (ESKD) confers a higher 5-year mortality (~50%) than any malignancy, except for lung (~80%) and pancreatic (~90%) cancers [1]. Yet the nephrology f ield had the lowest productivity in new drug approvals, largely due to the decades-long standing regulatory standards of demonstrating benefit in reaching doubling of serum creatinine, ESKD, or death (referred to as TRAD going forward), which are clinical endpoints requiring several years and large numbers of study participants to achieve statistical significance between active vs. comparator (usually placebo) arms. Conducting trials in rare kidney diseases, typically defined as a prevalence <200,000 or <50 in 100,000, has the additional challenge of small numbers of eligible patients.

Received Date: 2025-09-18 | Published Date: 2025-10-14