Virtually no therapeutic interventions besides proven dialysis treatment are available in properly designed randomised controlled trials (RCTs) for the improvement in outcomes.
According to a recent review of hemodialysis RCTs, among 10,713 outcome measures, surrogates such as phosphate, dialysis adequacy, anemia, inflammatory markers, and calcium were found to be the most common.
Very infrequently patient-centered outcomes such as mortality, cardiovascular disease, and quality of life were reported.
Clinical trials in end-stage renal disease—priorities and challenges review author professor Csaba Kovesdy said: “The ESRD population is diverse and complex, making it difficult to test interventions within the framework of a traditional RCT design that has resulted in unexpectedly low event rates and high drop-out and cross-over rates, rendering results internally invalid and yielding inconclusive results.
“The recent emergence of various RCT designs could aid in making ESRD clinical trials more successful.”
Pragmatic clinical trials (PCTs) were introduced to improve the external validity of clinical trials, by implementing enrolment criteria, clinically relevant comparators and evaluation of interventions within clinical practice.
PCTs are particularly feasible in the hemodialysis population with the use of electronic health records (EHRs) and the clustering of patients within dialysis units using uniform clinical practices.
Other emerging RCT designs such as adaptive platform designs are also available that could result in more successful testing of interventions in ESRD.
Kovesdy further added: “The application of such novel RCT designs could result in benefits including reduced trial cost, the examination of a broader, more representative population, and the testing of a higher number and more clinically relevant interventions, which is really needed.”