Economic Evaluations of Orphan Drugs for Rare Kidney Diseases in Low and Middle Income Countries: A Systematic Review and Bibliometric Analysis
Author(s)
Mohammed Alfaqeeh, MSc1, Auliya Abdurrohim Suwantika, PhD1, Maarten Jacobus Postma, PhD2, Rizkia Andicha Putra, BSc1, Jasmine Rani Aisyah, BSc1, Fima Perdani Rahayu, BSc1, Lubna Farhana, BSc1, Muhammad Ilyas, BSc1, Shofuro Sholihah, BSc1, Neily Zakiyah, PhD1.
1Universitas Padjadjaran, Bandung, Indonesia, 2University of Groningen, Groningen, Netherlands.
1Universitas Padjadjaran, Bandung, Indonesia, 2University of Groningen, Groningen, Netherlands.
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 (LMICs) over the past decade.
METHODS: A systematic review was conducted following PRISMA 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 LMICs, reporting outcomes such as Incremental Cost-Effectiveness Ratio (ICER) per Quality-Adjusted Life Years (QALYs) gained, Life Years (LYs) gained, and other clinical outcomes. Study quality was assessed using the CHEERS 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 LYs and QALYs 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), though 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 LMICs.
METHODS: A systematic review was conducted following PRISMA 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 LMICs, reporting outcomes such as Incremental Cost-Effectiveness Ratio (ICER) per Quality-Adjusted Life Years (QALYs) gained, Life Years (LYs) gained, and other clinical outcomes. Study quality was assessed using the CHEERS 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 LYs and QALYs 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), though 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 LMICs.
Conference/Value in Health Info
2025-09, ISPOR Real-World Evidence Summit 2025, Tokyo, Japan
Value in Health Regional, Volume 49S (September 2025)
Code
RWD153
Topic Subcategory
Reproducibility & Replicability
Disease
SDC: Urinary/Kidney Disorders