Scoping Review of Economic Analyses of Rare Kidney Diseases

Abstract

IntroductionRare kidney diseases (RKD) 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 RKD. MethodsWe conducted a systematic scoping review to identify economic evaluations, health technology assessments and cost-of-illness studies relating to RKD published since 2012. Results161 published studies, including 66 cost of illness studies, 95 economic evaluations and 72 grey literature reports were 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 RKD. 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 RKD. 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. ConclusionsWe found large gaps in the economic evidence base for RKD and limited adaption 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.