The societal economic burden of autosomal dominant polycystic kidney disease in the United States.

BACKGROUND: 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. 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). METHODS: A 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. RESULTS: The 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%). CONCLUSIONS: ADPKD 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.

Screening for intracranial aneurysms in autosomal dominant polycystic kidney disease is cost-effective.

Intracranial aneurysm rupture is a dramatic complication of autosomal dominant polycystic kidney disease (ADPKD). It remains uncertain whether screening should be widespread or only target patients with risk factors (personal or familial history of intracranial aneurysm), with an at-risk profession, or those who request screening. We evaluated this in a single-center cohort of 495 consecutive patients with ADPKD submitted to targeted intracranial aneurysm screening. Cerebral magnetic resonance angiography was proposed to 110 patients with a familial history of intracranial aneurysm (group 1), whereas it was not our intention to propose it to 385 patients without familial risk (group 2). Magnetic resonance angiography results, intracranial aneurysm prophylactic repair, rupture events, and cost-effectiveness of intracranial aneurysm screening strategies were retrospectively analyzed. During a median follow up of 5.9 years, five non-fatal intracranial aneurysm ruptures occurred (incidence rate 2.0 (0.87-4.6)/1000 patients-year). In group 1, 90% of patients were screened and an intracranial aneurysm was detected in 14, treated preventively in five, and ruptured in one patient despite surveillance. In group 2, 21% of patients were screened and an intracranial aneurysm was detected in five, and treated preventively in one. Intracranial aneurysm rupture occurred in four patients in group 2. Systematic screening was deemed cost-effective and provides a gain of 0.68 quality-adjusted life years compared to targeted screening. Thus, the intracranial aneurysm rupture rate is high in ADPKD despite targeted screening, and involves mostly patients without familial risk factors. Hence, cost-utility analysis suggests that intracranial aneurysm screening could be proposed to all ADPKD patients.

Real-world costs of autosomal dominant polycystic kidney disease in the Nordics.

BACKGROUND: There is limited real-world data on the economic burden of patients with autosomal dominant polycystic kidney disease (ADPKD). The objective of this study was to estimate the annual direct and indirect costs of patients with ADPKD by severity of the disease: chronic kidney disease (CKD) stages 1-3; CKD stages 4-5; transplant recipients; and maintenance dialysis patients. METHODS: A retrospective study of ADPKD patients was undertaken April-December 2014 in Denmark, Finland, Norway and Sweden. Data on medical resource utilisation were extracted from medical charts and patients were asked to complete a self-administered questionnaire. RESULTS: A total of 266 patients were contacted, 243 (91%) of whom provided consent to participate in the study. Results showed that the economic burden of ADPKD was substantial at all levels of the disease. Lost wages due to reduced productivity were large in absolute terms across all disease strata. Mean total annual costs were highest in dialysis patients, driven by maintenance dialysis care, while the use of immunosuppressants was the main cost component for transplant care. Costs were twice as high in patients with CKD stages 4-5 compared to CKD stages 1-3. CONCLUSIONS: Costs associated with ADPKD are significant and the progression of the disease is associated with an increased frequency and intensity of medical resource utilisation. Interventions that can slow the progression of the disease have the potential to lead to substantial reductions in costs for the treatment of ADPKD.

Is CAPD a viable option among ADPKD with end stage renal disease population in India? Its outcomes and economics.

Autosomal dominant polycystic kidney disease (ADPKD) is the most common inherited renal disease, with 50-75% of these patients requiring renal replacement therapy (RRT). The outcome of peritoneal dialysis (PD) in ADPKD with end-disease renal disease (ESRD) is not clearly defined, more so in developing countries. We conducted a retrospective analysis of the outcomes and economics of PD in these ESRD patients and compared them with other causes of ESRD on PD. Data were reviewed of all the PD patients who were followed-up at our institute from January 2007 to December 2011. The inclusion criteria were ADPKD patients who chose PD as the dialysis modality (Group 1), while age and gender-matched ESRD (other than ADPKD) patients who were started on PD during the same period were considered as the other group (Group 2). A total of 26 ADPKD patients underwent PD with an average size of kidneys among ADPKD ESRD patients of 15.2 + 2.1 cm. The overall peritonitis rates were similar among the compared groups. The median survival for the first peritonitis episodes were 1.2 and 1.8 years (95% confidence interval 0.82-1.91) for the control and ADPKD groups, respectively. The overall patient survival was 22 among PKD while five patients died among the control group. Among PKD, one patient died due to intra-cerebral bleed while one patient had severe cyst hemorrhage and infection, while three others had peritonitis and sepsis. Hernia was observed in four ADPKD patients, once on PD that was surgically corrected and PD was resumed in all. Two patients lost the catheter due to peritonitis while one patient had membrane failure while one underwent surgical exploration due to diverticulosis. PD treatment was not prevented by voluminous kidneys in any of these patients and no patient ceased PD treatment due to insufficient peritoneal space. Besides this, the cost on PD was much less as compared with that on hemodialysis (HD). PD is a reasonable mode of RRT among ADPKD, where HD is not possible or contraindicated with lesser risks to bleeding and infections, and the cost benefit favoring PD in general.

Hospital-based inpatient resource utilization associated with autosomal dominant polycystic kidney disease in the US.

OBJECTIVE: Polycystic kidney disease (PKD) is a clinically and genetically heterogeneous class of genetic disorders characterized by development of renal cysts leading to renal failure and end stage renal disease (ESRD). Autosomal dominant polycystic kidney disease (ADPKD) accounts for the majority of PKD cases and is the predominant monogenic cause of ESRD. Limited information on patient characteristics and healthcare resource utilization is available in this population. This study assessed hospital-based inpatient utilization of patients with ADPKD in the US to help further understand the disease, which may lead to treatments that delay progression and reduce healthcare resource utilization. METHODS: A cross-sectional analysis was conducted using MedAssets Health System Data to investigate inpatient resource utilization for a total of 1876 patients hospitalized with ADPKD or chronic kidney disease (CKD). Patient characteristics and inpatient resource utilization were compared between hospitalized patients with ADPKD and CKD, including demographic and clinical characteristics, overall health, rates of complications and surgical interventions, and average length of hospital and intensive care unit stay. RESULTS: Compared with patients with CKD, patients with ADPKD were more likely to have commercial insurance as their primary payer (36.1 vs 17.8%) and were significantly younger (mean age 57.9 vs 69.5 years) and generally healthier (Charlson Comorbidity Score of 2.0 vs 3.3). Patients with ADPKD also had a substantially shorter average length of hospital stay (6.3 vs 10.3 days). However, patients with ADPKD experienced more kidney-related complications and a higher surgical procedure rate (mainly for transplant and complete nephrectomy). CONCLUSIONS: Although patients with ADPKD were generally healthier than patients with CKD, specific kidney function complications were more frequent. Patients with ADPKD had a higher rate of major kidney procedures, which may contribute to the high burden of ADPKD-related hospital-based inpatient resource utilization.

Molecular diagnosis of autosomal dominant polycystic kidney disease.

Autosomal dominant polycystic kidney disease (ADPKD) is the most common inherited renal disorder. Its estimated prevalence is 1 per 800 individuals. ADPKD patients constitute 8% of the population on dialysis or kidney transplantation. The disease can be diagnosed using radiological or genetic procedures. Direct genetic diagnosis of the disease can now be performed in Spain; however, it is not an easy or cheap test. This is why every case should be considered individually to determine whether genetic testing is appropriate, and to determine which genetic test is most adequate. Genetic testing in ADPKD is of special interest for living donors and neonatal and sporadic cases. Genetic testing offers the chance of performing prenatal or pre-implantation testing of embryos in families with severe cases of the disease. Also, this will enable the disease to be treated, when specific treatment becomes available, in cases that would not be candidates for treatment without genetic confirmation.

Economic evaluation of prenatal diagnosis: a methodological review.

This paper discusses the role of economic evaluation in prenatal diagnosis. As the availability of new and improved techniques of prenatal diagnosis increases, so does the relevance of economic evaluation. The various methods of economic evaluation are discussed in the context of amniocentesis and chorionic villus sampling. It is argued that in view of the potentially wide range of benefits from prenatal diagnosis, more sophisticated measures of benefit should be incorporated into economic evaluations. Further development of utility-based measures and monetary valuation is suggested.

Insurance for autosomal dominant polycystic kidney disease patients prior to end-stage renal disease.

There recently has been substantial dialogue about access to health insurance for Americans. This discussion has highlighted the issues of pre-existing diseases and portability as barriers to adequate health insurance coverage. For these reasons we decided to investigate the issues relating to health insurance and life insurance coverage experienced by patients with autosomal dominant polycystic kidney disease (ADPKD). A questionnaire-based study was conducted. Two hundred thirty-eight of 354 subjects responded. There was no significant difference in gender, age, number of children, or level of renal function between responders and nonresponders. Twenty-eight of the 238 respondents had eight-stage renal disease and were eligible for Medicare; these patients were not used in the analyses relating to health insurance, but were used in the analyses relating to life insurance. Although 87% of the ADPKD patients were concerned about the availability of health insurance, 88% were currently insured. Eight-three percent of subjects with health insurance obtained it through their own or their spouse's employer. Of those individuals with employer-based health insurance who were aware of their ADPKD, only 25% informed their employer and 35% informed their insurer at the start of coverage. Fifty-seven percent of those with employer-based health insurance had this availability determine their job choice and 37% stayed in the job because of health insurance. Thirty percent of the subjects had previously been denied health insurance. Although subjects were less concerned about life insurance, many of the same types of issues and factors were present. Thus, the current lack of universal health care in this country creates anxiety and difficulties for patients with ADPKD. The effect of a pre-existing condition and the lack of portability resulted in denials, work choice limitation, and unwillingness to share health information for this patient population with a hereditary, systemic disease.