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Economic Evaluations of Orphan Drugs for Rare Kidney Diseases in Low- and Middle-Income Countries: A Bibliometric
Mohammed Alfaqeeh1, Auliya A Suwantika2, Maarten J Postma3
1Doctoral Program of Pharmacy, Faculty of Pharmacy, Universitas Padjadjaran, Bandung, Republic of Indonesia; Department of Pharmacology and Clinical Pharmacy, Faculty of Pharmacy, Universitas Padjadjaran, Bandung, Republic of Indonesia.
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.
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