Adrenocortical carcinoma (ACC) is an orphan disease lacking effective systemic treatment
options. The low incidence of the disease and high cost of clinical trials are major
obstacles in the search for improved treatment strategies. As a novel approach, registry-based
clinical trials have been introduced in clinical research, so allowing for significant
cost reduction, but without compromising scientific benefit. Herein, we describe how
the European Network for the Study of Adrenal Tumours (ENSAT) could transform its
current registry into one fit for a clinical trial infrastructure. The rationale to
perform randomized registry-based trials in ACC is outlined including an analysis
of relevant limitations and challenges. We summarize a survey on this concept among
ENSAT members who expressed a strong interest of the concept and rated its scientific
potential as high. Legal aspects, including ethical approval of registry-based randomization
were identified as potential obstacles. Finally, we describe three potential randomized
registry-based clinical trials in an adjuvant setting and for advanced disease with
a high potential to be executed within the framework of an advanced ENSAT registry.
Thus we therefore provide the basis for future registry-based trials for ACC patients.
This could ultimately provide proof-of-principle of how to perform more effective
randomized trials for an orphan disease.
