title: Creating an effective clinical registry for rare diseases.
abstract: The exposure of clinicians to patients with rare gastrointestinal diseases is limited. This hurts clinical studies, which impedes accumulation of scientific knowledge on the natural disease course, treatment outcomes and prognosis in these patients. An excellent method to detect patterns on an aggregate level that would not be possible to discover in individual cases, is a registry study. This paper aims to describe a template to create a successful international registry for rare diseases. We focus mainly on rare hepatic diseases, but lessons from this paper serve other fields in medicine, as well.
authors.affiliation: Department of Gastroenterology and Hepatology, Radboud University, Medical Centre Nijmegen, Nijmegen, The Netherlands.
authors.forename: Hedwig Ma
authors.initials: HM
authors.lastname: D'Agnolo
authors.affiliation: Radboud Institute for Health Sciences, Radboud University Medical Centre, Nijmegen, The Netherlands.
authors.forename: Wietske
authors.initials: W
authors.lastname: Kievit
authors.affiliation: Institute for Biomedical Research of Malaga (IBIMA), Virgen de la Victoria [Virgin of Victory] University Hospital and School of Medicine, Malaga, Spain; Instituto de Investigación Biomédica de Málaga-IBIMA, Hospital Universitario Virgen de la Victoria, Universidad de Málaga, Unidad de Gestión Clínica de Aparato Digestivo; Centro de Investigación Biomédica en Red de Enfermedades Hepáticas y Digestivas (CIBERehd. Málaga, Spain).
authors.forename: Raul J
authors.initials: RJ
authors.lastname: Andrade
authors.affiliation: Department of Transplantation Medicine; Division of Cancer Medicine, Surgery and Transplantation; Oslo University Hospital, Oslo, Norway.
authors.forename: Tom Hemming
authors.initials: TH
authors.lastname: Karlsen
authors.affiliation: Department of Gastroenterology, Hepatology and Endocrinology, Hannover Medical School, Hannover, Germany.
authors.forename: Heiner
authors.initials: H
authors.lastname: Wedemeyer
authors.affiliation: Department of Gastroenterology and Hepatology, Radboud University, Medical Centre Nijmegen, Nijmegen, The Netherlands.
authors.forename: Joost Ph
authors.initials: JP
authors.lastname: Drenth
keywords: Clinical registry; database; gastrointestinal disease; liver disease; liver transplantation; practical guide; rare diseases; rare liver disorders; registry design