Clinical trials in end-stage renal disease—priorities and challenges

Abstract

Patients with end-stage renal disease (ESRD) experience extremely high morbidity and mortality and there are virtually no therapeutic interventions besides dialysis treatment that are proven in properly designed randomized controlled trials (RCTs) to improve patients’ outcomes. Historically, the number of RCTs performed in the ESRD population has been very low compared with other medical subspecialties, and several of the few large RCTs have yielded inconclusive or negative results, dampening enthusiasm for future investment in similar trials. Recent initiatives promoting a focus on patient-centered outcomes and more active patient and caregiver involvement in the planning and conduct of clinical trials may result in more clinically relevant RCTs and broader participation from patients representing the diversity of the ESRD population. The adoption of novel clinical trial design elements characteristic of pragmatic clinical trials and platform trials could help improve both the internal and external validity of RCTs in ESRD, ultimately resulting in the adoption of therapeutic interventions that can be rapidly translated to clinical practice.