Drug Development in Kidney Disease: Proceedings From a Multistakeholder Conference

Abstract

Occasional bursts of discovery and innovation have appeared during the otherwise stagnant past several decades of drug development in nephrology. Among other recent drug discoveries, the unexpected kidney benefits observed with sodium/glucose cotransporter 2 inhibitors may herald a renaissance of drug development in kidney disease. This recent progress highlights the need to further promote and stimulate research and development of promising therapies that may ameliorate the morbidity and mortality associated with kidney disease. To help identify and address barriers to drug development in nephrology, the Duke Clinical Research Institute convened a conference in April 2019 that included stakeholders from academia, industry, government agencies, and patient advocacy. From these discussions, several opportunities were identified to improve every stage of drug development for kidney disease from early discovery to implementation into practice. Key topics reviewed in this article are the utility of interconnected data and site research networks, surrogate end points, pragmatic and adaptive trial designs, the promising uses of real-world data, and methods to improve the generalizability of trial results and uptake of approved drugs for kidney-related diseases.

Keywords: Drug development; administrative data sets; clinical investigation; clinical trial design; conference report; data integration; disease network; drug discovery; electronic health record (EHR); genetic determinants of disease; health care innovation; kidney disease end points; nephrology research; patient-centered endpoints (PCEs); post-approval trials; post-marketing studies; pragmatic trial; real-world evidence; surrogate end points; therapeutic target.