An Overview of the Evidence for the Clinical Practice Guidelines in Rare Disease

Article type
Authors
Shi Q1, Wang Z2, Zhou Q1, Zhao S3, Xun Y2, Chen Y4
1The First School of Clinical Medicine, Lanzhou University
2Evidence-Based Medicine Center, School of Basic Medical Sciences, Lanzhou University
3School of Public Health, Lanzhou University
4Evidence-Based Medicine Center, School of Basic Medical Sciences, Lanzhou University; WHO Collaborating Centre for Guideline Implementation and Knowledge Translation; Chinese GRADE Center; Cochrane China Network, Lanzhou
Abstract
Background: Rare diseases propose a global public health priority to health care practitioners and policy makers, and clinical practice guidelines that summarize evidence and use clear criteria to generate recommendations are necessary to support decision-making in this field. However, there are numerous barriers to evidence synthesize and generation in rare diseases due to the lack of high certainty evidence. Strategies may include thought use of indirect evidence, used of systematic observation forms, or the use of registry data, etc. Thus, understanding the evidence status used in the clinical practice guidelines in rare diseases is essential for follow-up research.

Objectives: To investigate the evidence overview of the clinical practice guidelines for rare diseases.

Methods: We conducted a scoping review on clinical practice guidelines for rare diseases. A systematic search on databases (PubMed, Embase and Orphanet), relevant guideline websites, and government health agency websites from their inception to April, 2020 was performed and we selected clinical practice guidelines related to rare disease. No language restriction was applied. Two researchers independently screened the records and extracted data, disagreements resulted through discussion or by a third party. Data processing was performed by Stata statistical software 14 to present descriptive results.

Results: The results will be presented at the meeting.

Conclusions: The results will be presented at the meeting.

Patient or healthcare consumer involvement: None