@article{3076457,
    title = "ENSAT registry-based randomized clinical trials for adrenocortical carcinoma",
    author = "Crona, J. and Baudin, E. and Terzolo, M. and Chrisoulidou, A. and Angelousi, A. and Ronchi, C.L. and Oliveira, C.L. and Nieveen van Dijkum, E.J.M. and Ceccato, F. and Borson-Chazot, F. and Reimondo, G. and Tiberi, G.A.M. and Ettaieb, H. and Kiriakopoulos, A. and Letizia, C. and Kastelan, D. and Osher, E. and Yiannakopoulou, E. and Arnaldi, G. and Assié, G. and Paiva, I. and Bourdeau, I. and Newell-Price, J. and Nowak, K.M. and Tous Romero, M. and de Martino, M.C. and Bugalho, M.J. and Sherlock, M. and Vantyghem, M.-C. and Dennedy, M.C. and Loli, P. and Rodien, P. and Feelders, R. and de Krijger, R. and van Slycke, S. and Aylwin, S. and Morelli, V. and Vroonen, L. and Shafigullina, Z. and Bancos, I. and Trofimiuk-Müldner, M. and Quinkler, M. and Luconi, M. and Kroiss, M. and Naruse, M. and Igaz, P. and Mihai, R. and della Casa, S. and Berruti, A. and Fassnacht, M. and Beuschlein, F.",
    journal = "European Journal of Endocrinology",
    year = "2021",
    volume = "184",
    number = "2",
    pages = "R51-R59",
    publisher = "BioScientifica Ltd",
    issn = "0804-4643, 1479-683X",
    doi = "10.1530/EJE-20-0800",
    keywords = "cisplatin;  etoposide;  mitotane, adjuvant chemotherapy;  adrenal cortex carcinoma;  adrenal tumor;  advanced cancer;  cancer registry;  clinical protocol;  comparative study;  drug efficacy;  European Organization for Research and Treatment of Cancer Quality of Life Questionnaire Core 30;  human;  legal aspect;  online monitoring;  overall survival;  priority journal;  progression free survival;  randomized controlled trial (topic);  rare disease;  reliability;  Review;  treatment response;  validity;  adrenal cortex carcinoma;  adrenal cortex tumor;  endocrinology;  Europe;  evidence based medicine;  organization and management;  procedures;  register;  social network, Adrenal Cortex Neoplasms;  Adrenocortical Carcinoma;  Endocrinology;  Europe;  Evidence-Based Medicine;  Humans;  Randomized Controlled Trials as Topic;  Registries;  Social Networking",
    abstract = "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 in 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. © 2021 BioScientifica Ltd.. All rights reserved."
}