Abstract

Objectives

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.

Methods

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 Economic Evaluation Reporting Standards checklist. Bibliometric analysis was performed with VOSviewer and Tableau.

Results

Among 406 identified studies, 16 met inclusion criteria. Cost-utility analysis was most common (88%), followed by cost-effectiveness (6%) and cost-minimization (6%). Most studies adopted a healthcare system (56%) or societal (19%) perspective. The majority used life-years and quality-adjusted life-years as outcome measures and considered direct medical costs, with 63% applying both probabilistic and deterministic sensitivity analyses. Reporting quality was high (87.5% rated as good), although gaps remained in heterogeneity, distributional effects, and funding disclosure. Overall, 63% found orphan drugs for RKDs cost-effective. Bibliometric analysis highlighted themes such as “cost-effectiveness,” “advanced renal cell carcinoma,” and “nivolumab.” Geographically, 67% of studies were conducted in China.

Conclusions

This review unexpectedly found that many studies identified orphan drugs for RKDs as cost-effective, despite their traditionally high costs. Future evaluations should refine methods to better capture long-term value and affordability in low- and middle-income countries.