Creating an effective clinical registry for rare diseases. Issue 3 (June 2016)

• Record Type: Journal Article
• Authors: D'Agnolo, Hedwig MA; Kievit, Wietske; Andrade, Raul J; Karlsen, Tom Hemming; Wedemeyer, Heiner; Drenth, Joost PH
• Is Part Of: United European Gastroenterology journal. Volume 4:Issue 3(2016:Jun.)
• Page Start: 333
• Page End: 338
• Publication Date: 2016-06
• Subjects: Clinical registry -- database -- gastrointestinal disease -- liver disease -- liver transplantation -- practical guide -- rare diseases -- rare liver disorders -- registry design; Gastroenterology -- Periodicals; Periodicals; 616.33005
• DOI: 10.1177/2050640615618042
• Languages: English
• ISSNs: 2050-6406

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.