Scoping Review of Economic Analyses of Rare Kidney Diseases
Introduction: Rare kidney diseases (RKDs) place a substantial economic burden on patients and health systems, the extent of which is unknown and may be systematically underestimated by health economic techniques. We aimed to investigate the economic burden and cost-effectiveness evidence base for RK...
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Elsevier
2024-12-01
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| Series: | Kidney International Reports |
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| Online Access: | http://www.sciencedirect.com/science/article/pii/S2468024924019223 |
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| author | Blake Angell Siyuan Wang Thomas Gadsden Monica Moorthy Charu Malik Jonathan Barratt Olivier Devuyst Ifeoma I. Ulasi Daniel P. Gale Agnivo Sengupta Anna Palagyi Vivekanand Jha Stephen Jan |
| author_facet | Blake Angell Siyuan Wang Thomas Gadsden Monica Moorthy Charu Malik Jonathan Barratt Olivier Devuyst Ifeoma I. Ulasi Daniel P. Gale Agnivo Sengupta Anna Palagyi Vivekanand Jha Stephen Jan |
| author_sort | Blake Angell |
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| description | Introduction: Rare kidney diseases (RKDs) place a substantial economic burden on patients and health systems, the extent of which is unknown and may be systematically underestimated by health economic techniques. We aimed to investigate the economic burden and cost-effectiveness evidence base for RKDs. Methods: We conducted a systematic scoping review to identify economic evaluations, health technology assessments, and cost-of-illness studies relating to RKDs, published since 2012. Results: A total of 161 published studies, including 66 cost-of-illness studies and 95 economic evaluations; 72 grey literature reports were also included. Most published literature originated from high-income nations, particularly the USA (81 studies), and focused on a handful of diseases, notably renal cell carcinomas (70) and systemic lupus erythematosus (36). Limited evidence was identified from lower-income settings and there were few studies of genetic conditions, which make up most RKDs. Some studies demonstrated the cost-effectiveness of existing treatments; however, there were limited considerations of broader economic impacts on patients that may be important to those with RKDs. Included health technology assessments highlighted difficulties in obtaining high-quality clinical evidence for treatments in very small patient populations, and often considered equity issues and other patient impacts qualitatively alongside clinical and economic evidence in their recommendations. Conclusion: We found large gaps in the economic evidence base for RKDs and limited adaptation of methods to account for the uniqueness of these diseases. There may be significant scope for innovation in building an investment case for RKD treatments, as well as in decision-making processes to inform investment decisions. |
| format | Article |
| id | doaj-art-07e69a01cf5d4b479a22e848f6ed2eeb |
| institution | OA Journals |
| issn | 2468-0249 |
| language | English |
| publishDate | 2024-12-01 |
| publisher | Elsevier |
| record_format | Article |
| series | Kidney International Reports |
| spelling | doaj-art-07e69a01cf5d4b479a22e848f6ed2eeb2025-08-20T02:33:36ZengElsevierKidney International Reports2468-02492024-12-019123553356910.1016/j.ekir.2024.09.004Scoping Review of Economic Analyses of Rare Kidney DiseasesBlake Angell0Siyuan Wang1Thomas Gadsden2Monica Moorthy3Charu Malik4Jonathan Barratt5Olivier Devuyst6Ifeoma I. Ulasi7Daniel P. Gale8Agnivo Sengupta9Anna Palagyi10Vivekanand Jha11Stephen Jan12The George Institute for Global Health, University of New South Wales, Sydney, Australia; Correspondence: Blake Angell, The George Institute for Global Health, Level 18 International Tower 3, 300 Barangaroo Avenue, Sydney 2000, Australia.The George Institute for Global Health, University of New South Wales, Sydney, AustraliaThe George Institute for Global Health, University of New South Wales, Sydney, AustraliaInternational Society of Nephrology, Brussels, BelgiumInternational Society of Nephrology, Brussels, BelgiumDepartment of Cardiovascular Sciences, University of Leicester, Leicester, UKDepartment of Physiology, Mechanisms of Inherited Kidney Disorders, University of Zurich, Zurich, Switzerland; Division of Nephrology, Cliniques Universitaires Saint-Luc, UCLouvain, Brussels, BelgiumRenal Unit, Department of Medicine, College of Medicine, University of Nigeria, Ituku-Ozalla, Enugu, Nigeria; Renal Unit, Department of Medicine, University of Nigeria Teaching Hospital, Ituku-Ozalla, Enugu, Nigeria; Renal Unit, Department of Internal Medicine, Alex Ekwueme Federal University Teaching Hospital, Abakaliki, NigeriaNational Registry of Rare Kidney Diseases, Bristol, UK; Department of Renal Medicine, University College London, London, UKThe George Institute for Global Health, University of New South Wales, Sydney, AustraliaThe George Institute for Global Health, University of New South Wales, Sydney, AustraliaInternational Society of Nephrology, Brussels, Belgium; The George Institute for Global Health, University of New South Wales, New Delhi, India; School of Public Health, Imperial College, London, UK; Prasanna School of Public Health, Manipal Academy of Higher Education, Manipal, IndiaThe George Institute for Global Health, University of New South Wales, Sydney, AustraliaIntroduction: Rare kidney diseases (RKDs) place a substantial economic burden on patients and health systems, the extent of which is unknown and may be systematically underestimated by health economic techniques. We aimed to investigate the economic burden and cost-effectiveness evidence base for RKDs. Methods: We conducted a systematic scoping review to identify economic evaluations, health technology assessments, and cost-of-illness studies relating to RKDs, published since 2012. Results: A total of 161 published studies, including 66 cost-of-illness studies and 95 economic evaluations; 72 grey literature reports were also included. Most published literature originated from high-income nations, particularly the USA (81 studies), and focused on a handful of diseases, notably renal cell carcinomas (70) and systemic lupus erythematosus (36). Limited evidence was identified from lower-income settings and there were few studies of genetic conditions, which make up most RKDs. Some studies demonstrated the cost-effectiveness of existing treatments; however, there were limited considerations of broader economic impacts on patients that may be important to those with RKDs. Included health technology assessments highlighted difficulties in obtaining high-quality clinical evidence for treatments in very small patient populations, and often considered equity issues and other patient impacts qualitatively alongside clinical and economic evidence in their recommendations. Conclusion: We found large gaps in the economic evidence base for RKDs and limited adaptation of methods to account for the uniqueness of these diseases. There may be significant scope for innovation in building an investment case for RKD treatments, as well as in decision-making processes to inform investment decisions.http://www.sciencedirect.com/science/article/pii/S2468024924019223cost-effectivenesseconomic burdenrare kidney disease |
| spellingShingle | Blake Angell Siyuan Wang Thomas Gadsden Monica Moorthy Charu Malik Jonathan Barratt Olivier Devuyst Ifeoma I. Ulasi Daniel P. Gale Agnivo Sengupta Anna Palagyi Vivekanand Jha Stephen Jan Scoping Review of Economic Analyses of Rare Kidney Diseases Kidney International Reports cost-effectiveness economic burden rare kidney disease |
| title | Scoping Review of Economic Analyses of Rare Kidney Diseases |
| title_full | Scoping Review of Economic Analyses of Rare Kidney Diseases |
| title_fullStr | Scoping Review of Economic Analyses of Rare Kidney Diseases |
| title_full_unstemmed | Scoping Review of Economic Analyses of Rare Kidney Diseases |
| title_short | Scoping Review of Economic Analyses of Rare Kidney Diseases |
| title_sort | scoping review of economic analyses of rare kidney diseases |
| topic | cost-effectiveness economic burden rare kidney disease |
| url | http://www.sciencedirect.com/science/article/pii/S2468024924019223 |
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