Should We Still Believe in Randomized Controlled Trials in Nephrology?

Area of Science:

• Nephrology
• Clinical Trials

Background:

• Randomized controlled trials (RCTs) are vital for clinical decisions in nephrology.
• Previous RCTs established the renoprotective benefits of renin-angiotensin system inhibitors for proteinuric nephropathies.

Purpose of the Study:

• To highlight concerns regarding the increasing cost, complexity, and duration of clinical studies.
• To identify critical flaws in recent nephrology RCTs that hindered definitive conclusions.

Main Methods:

• Review of pivotal and recent large RCTs in nephrology.
• Analysis of investigational strategies, including dosing, control groups, patient selection, adverse event reporting, and outcome evaluation.

Main Results:

• Despite demonstrating treatment benefits, RCTs face challenges like high costs and complexity.
• Recent large RCTs failed to yield definitive conclusions due to issues such as fixed dosing, inappropriate controls, low-risk enrollment, poor adverse event reporting, and unreliable renal function assessment.

Conclusions:

• Identified biases in current RCTs necessitate a reevaluation of their design.
• Future nephrology RCTs require improved strategies for design, patient selection, and implementation to ensure reliable and definitive outcomes.