Abstract

BackgroundAutosomal dominant polycystic kidney disease (ADPKD) is one of the most common inherited kidney diseases characterized by progressive development of renal cysts and numerous extra-renal manifestations, eventually leading to kidney failure. Given its chronic and progressive nature, ADPKD is expected to carry a substantial economic burden over the course of the disease. However, there is a paucity of evidence on the impact of ADPKD from a societal perspective. This study aimed to estimate the direct and indirect costs associated with ADPKD in the United States (US).MethodsA prevalence-based approach using data from scientific literature, and governmental and non-governmental organizations was employed to estimate direct healthcare costs (i.e., medical services, prescription drugs), direct non-healthcare costs (i.e., research and advocacy, donors/recipients matching for kidney transplants, transportation to/from dialysis centers), and indirect costs (i.e., patient productivity loss from unemployment, reduced work productivity, and premature mortality, caregivers’ productivity loss and healthcare costs). The incremental costs associated with ADPKD were calculated as the difference between costs incurred over a one-year period by individuals with ADPKD and the US population. Sensitivity analyses using different sources and assumptions were performed to assess robustness of estimates and account for variability in published estimates.ResultsThe estimated total annual costs attributed to ADPKD in 2018 ranged from $7.3 to $9.6 billion in sensitivity analyses, equivalent to $51,970 to $68,091 per individual with ADPKD. In the base scenario, direct healthcare costs accounted for $5.7 billion (78.6%) of the total $7.3 billion costs, mostly driven by patients requiring renal replacement therapy ($3.2 billion; 43.3%). Indirect costs accounted for $1.4 billion (19.7%), mostly driven by productivity loss due to unemployment ($784 million; 10.7%) and reduced productivity at work ($390 million; 5.3%). Total excess direct non-healthcare costs were estimated at $125 million (1.7%).ConclusionsADPKD carries a considerable economic burden, predominantly attributed to direct healthcare costs, the majority of which are incurred by public and private healthcare payers. Effective and timely interventions to slow down the progression of ADPKD could substantially reduce the economic burden of ADPKD.

Highlights

  • Autosomal dominant polycystic kidney disease (ADPKD) is one of the most common inherited kidney diseases characterized by progressive development of renal cysts and numerous extra-renal manifestations, eventually leading to kidney failure

  • The incremental costs associated with ADPKD were estimated based on the average cost difference between an individual with ADPKD and an individual from the United States (US) population, and constitute the “excess” costs attributed to ADPKD; similar methodology has been used in several previous publications [18,19,20,21,22,23,24,25,26]

  • The economic burden associated with ADPKD for these individuals was estimated at $7.3 billion in 2018, that is $51,970 per individual with ADPKD (Table 1)

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Summary

Introduction

Autosomal dominant polycystic kidney disease (ADPKD) is one of the most common inherited kidney diseases characterized by progressive development of renal cysts and numerous extra-renal manifestations, eventually leading to kidney failure. This study aimed to estimate the direct and indirect costs associated with ADPKD in the United States (US). Autosomal dominant polycystic kidney disease (ADPKD) is the most common of the inherited renal cystic diseases, a group of related but pathologically distinct disorders characterized by the progressive development of renal cysts [1]. Given that ADPKD is a life-long and progressive disorder, a substantial economic burden over the course of the disease is expected, including a variety of treatmentrelated and other costs [6,7,8,9,10,11,12,13]. The only study that estimated both direct and indirect costs associated with ADPKD was based on a Nordic population (i.e., Denmark, Finland, Norway, and Sweden) [9], and may not be representative of the economic burden of ADPKD in the US

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