Nephrology has long been a difficult space for drug development: trials were slow, complex, high cost, and often unrewarding, constrained by fragmented care, blunt endpoints, noisy signals, and overburdened patients, even as regulators and investors demand clearer benefit and renal safety.

The field is now being rewritten by a narrowed focus to a defined set of rare renal indications, embracing strategic and tactical biomarker use, and reimagining where and how global trials are run, from patient‑centric designs to high‑prevalence regions and more coherent regulatory strategies. As a result, over the last decade or so, nephrology research has seen a shift away from large, high-cost chronic kidney disease (CKD) and dialysis programs toward a more targeted focus on a smaller set of rare nephrology indications, particularly IgA nephropathy, Focal Segmental Glomerulosclerosis (FSGS), Autosomal Dominant Polycystic Kidney Disease (ADPKD) and Alport Syndrome.

Trials have become more focused and aligned, with clearer control arms and more deliberate use of surrogate endpoints. These trials are increasingly conducted in regions where prevalence and enrollment potential are highest, particularly parts of APAC such as South Korea, China, Taiwan, and Singapore, while incorporating more patient input to improve feasibility and retention.

The result is a nephrology landscape that is more focused in its indication scope and delivers strategic, agile studies and takeaways that are better aligned with the lived realities of patients.

The changing regulatory landscape

Nephrology now operates in a regulatory environment shaped by years of fragmented clinical care and inconsistent trial practice, argues Jonathan Kornstein, Vice President and Therapeutic Area Head, Rare Disease and Pediatrics, Caidya. This has historically translated into a lack of “coordinated and standardized, harmonized approaches across all the regulatory bodies,” says Kornstein.

As sponsors pivot into more tightly defined rare nephrology indications, Kornstein believes regulators can enable greater alignment because indications and trial designs are clearer, and so can build programs that meet regional requirements without reinventing the trial each time. For sponsors, this means early endpoint dialogue and protocols that accommodate regional care differences while supporting a global submission strategy.

Innovations in trial designs

A key innovation in nephrology over the past decade has been a deliberate shift toward trial designs that prioritize the patient journey. As Kornstein explains: “Studies are [now] being designed around the patient rather than the data, with every effort made to keep burden low and make participation as easy as possible for both patients and caregivers,” noting that retention becomes a design principle, not an afterthought.

A related development is the growing incorporation of patient advocacy groups into study planning. Sponsors now seek input from these groups on design and logistics, which helps align protocols with what patients can realistically sustain over long follow-up periods.

Geographical shifts in research

Increasingly, nephrology sponsors are looking beyond traditional Western markets and orienting trials toward “where the patients are.” Kornstein highlights a sustained shift into the Asia–Pacific region, noting that for both earlier CKD/dialysis work and today’s rare nephrology studies, these regions offer higher prevalence for key renal indications and significantly higher enrollment rates than many Western countries.

This is not just about volume, but strategy: by prioritizing relatively untapped regions with less trial competition and stronger recruitment performance, sponsors can accelerate startup, de‑risk enrollment, and run programs more efficiently in a crowded global landscape.

Over time, biomarkers have become increasingly central to how the field understands renal disease progression and treatment efficacy. However, Dr Henry Cremisi, Executive Medical Director, Medical Affairs at Caidya, says that: “We are 20 years behind where oncology is in the use of biomarkers.”

Renal and cardiovascular‑renal‑metabolic (CVRM) biomarkers are shifting from peripheral scientific readouts to core strategic tools. Traditional endpoints like eGFR slope, creatinine, and proteinuria are essential but slow and nonspecific. In contrast, mechanistic biomarkers that track podocyte, tubular, vascular, complement, fibrotic, and immune pathways can reveal target engagement, early on‑target benefit, and emerging safety issues months or years sooner[i]. 

When deliberately aligned with mechanism, trial design, regulatory dialogue, and commercial planning, these biomarkers move from “interesting data” to decision‑shaping insights that accelerate learning, reduce uncertainty, and expand strategic options.

Cremisi argues that the real power of biomarkers lies in Phase II dose-finding, where they can fundamentally reshape how nephrology drugs are optimized. He cites ACE inhibitors as a cautionary example: they were originally approved on the basis of blood-pressure lowering alone, and only later did it become clear that the doses needed to control blood pressure are very different from those required to reduce proteinuria. Without biomarker-guided dose selection, he warns, sponsors risk locking in pivotal-stage doses that systematically underuse a drug’s renal benefits, effectively capping its impact before the program ever reaches Phase III.

Well-designed biomarker programs do more than fine-tune dose; they clarify mechanism of action, revealing whether a therapy simply halts damage or actively drives healing and resilience. Cremisi notes that they can also uncover “other indications for your medication that could be renal or extra renal,” turning early-phase studies into engines for future therapeutic discovery. Ultimately, strategic exploratory biomarkers are enhancing value by informing the product life cycle and future indications and revenue streams.

Solutions and opportunities

Kornstein argues that one of the clearest levers for improving trial speed and efficiency is to run nephrology studies where the epidemiology informs the patients. Sponsors, he says, too often default to regions “where studies have been done in the past,” instead of looking to higher‑prevalence, underutilized markets. By deliberately shifting into these less crowded areas with more eligible patients, they can accelerate recruitment and ease competition for participants.

Unlocking this opportunity, Kornstein says, requires disciplined, forward‑looking global planning. The most successful sponsors begin shaping their global execution nine to twelve months in advance, particularly for regions with longer regulatory timelines but stronger enrollment. Early alignment on country mix, regulatory strategy, and start‑up sequencing allows these high‑yield markets to come online alongside, rather than after, traditional Western sites.

However, a global strategy cannot be a generic one-size-fits-all approach. Sponsors need protocols flexible enough to reflect local standards of care, access to dialysis or specialist services, and practical realities for patients and sites. Strategic CRO partnerships are central here: bringing partners in early to advise on patient‑centric design, feasibility, and country selection turns regulatory and operational variability from a source of delay into a competitive advantage in delivering nephrology trials faster and more effectively.

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[i] Renal and CVRM Biomarkers: No longer just scientific tools, but strategic accelerators Henry D. Cremisi, MD Executive Medical Director, Caidya Jan 2026