Orphanet J Rare Dis
October 2024
We aim to illustrate the role of complete and transparent reporting coupled with access to data sourced from published systematic reviews, especially assisting in the identification of evidence for subgroups within the context of a rare disease. To accomplish this principle, we provide a real-world example encountered during the revision of the Dutch clinical practice guideline for hepatocellular carcinoma. Specifically, we retrieved insights from two Cochrane reviews to identify direct evidence concerning the diagnostic test accuracy of computed tomography and magnetic resonance imaging for detecting hepatocellular carcinomas in suspected patients without liver cirrhosis.
View Article and Find Full Text PDFWe aimed to develop and test a tool based on the re-weighted range voting mechanism to prioritize items (i.e. key questions) in a priority-setting assessment for clinical practice guidelines.
View Article and Find Full Text PDFObjective: For Dutch medical guidelines, Dutch research articles published in the NTvG (NederlandsTijdschriftvoorGeneeskunde) and other medical journals are not searched systematically and are only used sporadically. Using these publications in the process of guideline development can be useful for recommendations regarding the Dutch context of care. In this research, we have investigated how often and in which parts of Dutch guidelines articles published in NTvG are used.
View Article and Find Full Text PDFBackground: Goal Attainment Scaling (GAS) is an instrument that is intended to evaluate the effect of an intervention by assessing change in daily life activities on an individual basis. However, GAS has not been validated adequately in an RCT setting. In this paper we propose a conceptual validation plan of GAS in the setting of rare disease drug trials, and describe a hypothetical trial where GAS could be validated.
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