Introduction: Rare diseases (RDs) are often chronic and progressive life-threatening medical conditions that affect a low percentage of the population compared with other diseases. These conditions can be treated with medications known as orphan drugs (ODs). Unfortunately, there is no universal definition of RDs or ODs. This systematic review (SR) will identify the quantitative and qualitative criteria and the underlying rationale used internationally to define RDs and ODs.
Methods And Analysis: This protocol follows the conventions for the Preferred Reporting Items for Systematic Review and Meta-Analysis Protocols (2015 guidelines). A SR will be conducted, including a search of the following databases: PubMed, MEDLINE, EMBASE, Scopus, Web of Science, GreyLit and OpenGrey. Eligible publications will be selected based on predetermined inclusion criteria. Extracted data will be analysed using thematic and content analyses of qualitative descriptors, whereas quantitative data will be analysed descriptively and reported in the form of frequencies and percentages.
Ethics And Dissemination: No ethical approval is required since this SR focuses on the secondary analysis of data retrieved from the scientific literature. The outcomes of this SR will be published as part of a PhD thesis, presented at conferences, and published in peer-reviewed journals.
Prospero Registration Number: CRD42021252701.
Download full-text PDF |
Source |
|---|---|
| http://www.ncbi.nlm.nih.gov/pmc/articles/PMC9345065 | PMC |
| http://dx.doi.org/10.1136/bmjopen-2022-062126 | DOI Listing |
Publication Analysis
Top Keywords
Similar Publications
Circulating level of growth-differentiation factor 15 and the functional outcome after acute ischemic stroke: a systematic review and meta-analysis.
Head Face Med
December 2024
The Second Department of Neurology, The First People's Hospital of Nanning, No. 90, Qixing Road, Nanning, Guangxi Province, 530022, China.
Growth-differentiation factor 15 (GDF-15) is a cytokine involved in cellular stress responses and inflammation. This meta-analysis evaluates the association between circulating GDF-15 levels and functional outcomes in patients with acute ischemic stroke (AIS). A comprehensive search of Medline, Web of Science, Embase, Wanfang, and CNKI was conducted up to July 15, 2024.
View Article and Find Full Text PDFClassification of influencing factors of speaking-up behaviour in hospitals: a systematic review.
BMC Health Serv Res
December 2024
Centre for Safety in Healthcare, at the Faculty of Technology, Policy and Management, Delft University of Technology, Jaffalaan 5, Delft, 2628 BX, the Netherlands.
Background: Speaking up among healthcare professionals plays an essential role in improving patient safety and quality of care, yet it remains complex and multifaceted behaviour. Despite awareness of potential risks and adverse outcomes for patients, professionals often hesitate to voice concerns due to various influencing factors. This complexity has encouraged research into the determinants of speaking-up behaviour in hospital settings.
View Article and Find Full Text PDFEffect of perioperative dexmedetomidine on recovery of postoperative gastrointestinal function in patients with general anesthesia: a systematic review and meta-analysis.
BMC Anesthesiol
December 2024
Department of Anesthesiology, The First People's Hospital of Foshan, Lingnan Road 81#, Foshan City, 528000, China.
Background: There is controversy surrounding the influence of dexmedetomidine on the recovery of postoperative gastrointestinal dysfunction in patients under general anesthesia. The main purpose of this meta-analysis is to evaluate the effect of dexmedetomidine administration during the perioperative period on the recovery of gastrointestinal function in patients under general anesthesia.
Methods: A systematic review and meta-analysis with trial sequential analysis was performed to identify randomized controlled trials comparing dexmedetomidine administration with placebo for the recovery of gastrointestinal function.
Bias in machine learning applications to address non-communicable diseases at a population-level: a scoping review.
BMC Public Health
December 2024
Upstream Lab, MAP Centre for Urban Health Solutions, Li Ka Shing Knowledge Institute, Unity Health Toronto, 30 Bond Street, Toronto, ON, M5B 1W8, Canada.
Background: Machine learning (ML) is increasingly used in population and public health to support epidemiological studies, surveillance, and evaluation. Our objective was to conduct a scoping review to identify studies that use ML in population health, with a focus on its use in non-communicable diseases (NCDs). We also examine potential algorithmic biases in model design, training, and implementation, as well as efforts to mitigate these biases.
View Article and Find Full Text PDFPrevalence and risk factors associated with birth asphyxia among neonates delivered in China: a systematic review and meta-analysis.
BMC Pediatr
December 2024
Department of Nursing, Shenzhen People's Hospital (The Second Clinical Medical College, Jinan University; The First Affiliated Hospital, Southern University of Science and Technology), Shenzhen, Guangdong, China.
Background: Birth asphyxia is a critical condition caused by an insufficient oxygen supply during delivery, and it poses a major threat to the health of newborns. The present meta-analysis aimed to estimate the prevalence of birth asphyxia among neonates and identify its risk factors in China.
Methods: PubMed, EMBASE, Scopus, Web of Science, the China Academic Journals (CNKI), the Chinese Biomedical Literature (CBM), the China Science and Technology Journal Database (VIP), and the WanFang database were searched for related publications.
Want AI Summaries of new PubMed Abstracts delivered to your In-box?
Enter search terms and have AI summaries delivered each week - change queries or unsubscribe any time!