Economic Evaluations of Orphan Drugs for Rare Kidney Diseases in Low- and Middle-Income Countries: A Bibliometric Systematic Review With Policy and Evidence Gaps Analysis.
WHY IT MATTERS
If you have a rare kidney disease in a low- or middle-income country, this research highlights why your access to orphan drugs is limited — there's a major gap in evidence about whether these treatments are affordable and effective in your region.
Researchers looked at studies from 2014 to 2024 about the cost and value of special medicines for rare kidney diseases in poorer countries. They found that there isn't much research on whether these expensive medicines are worth the money in these areas. The study shows that doctors and governments need better information to decide if patients should have access to these treatments.
Economic Evaluations of Orphan Drugs for Rare Kidney Diseases in Low- and Middle-Income Countries: A Bibliometric Systematic Review With Policy and Evidence Gaps Analysis. Abstract: We conducted a systematic review and bibliometric analysis to explore the economic evaluation methods, outcomes, trends, and geographical distribution of orphan drugs for rare kidney diseases (RKDs) in low- and middle-income countries over the past decade. A systematic review was conducted following Preferred Reporting Items for Systematic Reviews and Meta-Analyses guidelines. Studies published from 2014 to 2024 were identified in PubMed and Scopus using keywords related to orphan drugs and RKDs. Eligible studies included full economic evaluations in low- and middle-income countries, reporting outcomes, such as incremental cost-effectiveness ratio per quality-adjusted life-years gained, life-years gained, and other clinical outcomes. Study quality was assessed using the Consolidated Health Authors: Alfaqeeh et al. Journal: Value in health : the journal of the International Society for Pharmacoeconomics and Outcomes Research MeSH: Humans, Orphan Drug Production, Developing Countries, Cost-Benefit Analysis, Rare Diseases, Quality-Adjusted Life Years, Kidney Diseases, Bibliometrics, Evidence Gaps