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Review
. 2016 Jun;4(3):333-8.
doi: 10.1177/2050640615618042. Epub 2015 Nov 13.

Creating an effective clinical registry for rare diseases

Hedwig Ma D'Agnolo  1 , Wietske Kievit  2 , Raul J Andrade  3 , Tom Hemming Karlsen  4 , Heiner Wedemeyer  5 , Joost Ph Drenth  1
Affiliations
Review

Creating an effective clinical registry for rare diseases

Hedwig Ma D'Agnolo et al. United European Gastroenterol J. 2016 Jun.

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.

Keywords: Clinical registry; database; gastrointestinal disease; liver disease; liver transplantation; practical guide; rare diseases; rare liver disorders; registry design.

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References

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