TY - JOUR
T1 - Creating an effective clinical registry for rare diseases
AU - D’Agnolo, Hedwig M. A.
AU - Kievit, Wietske
AU - Andrade, Raul J.
AU - Karlsen, Tom Hemming
AU - Wedemeyer, Heiner
AU - Drenth, Joost P. H.
PY - 2016/6/1
Y1 - 2016/6/1
N2 - 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.
AB - 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.
UR - https://www.scopus.com/inward/record.uri?partnerID=HzOxMe3b&scp=85006190463&origin=inward
U2 - https://doi.org/10.1177/2050640615618042
DO - https://doi.org/10.1177/2050640615618042
M3 - Review article
SN - 2050-6406
VL - 4
SP - 333
EP - 338
JO - United European gastroenterology journal
JF - United European gastroenterology journal
IS - 3
ER -