Cost-Effectiveness Analysis of Nefecon versus Best Supportive Care for People with Immunoglobulin A Nephropathy (IgAN) in the United States

References

• Lafayette RA, Kelepouris E. Immunoglobulin A nephropathy: advances in understanding of pathogenesis and treatment. Am J Nephrol. 2018;47(suppl1):43–52. doi:10.1159/000481636
   PubMedGoogle Scholar
• Wyatt RJ, Julian BA. IgA nephropathy. N Engl J Med. 2013;368(25):2402–2414. doi:10.1056/NEJMra1206793
   PubMed Web of Science ®Google Scholar
• McGrogan A, Franssen CF, de Vries CS. The incidence of primary glomerulonephritis worldwide: a systematic review of the literature. Nephrol Dial Transplant. 2011;26(2):414–430. doi:10.1093/ndt/gfq665
   PubMed Web of Science ®Google Scholar
• Kwon CS, Daniele P, Forsythe A, Ngai C. A systematic literatureA review of the epidemiology, health-related quality of life impact, and economic burden of immunoglobulin a nephropathy. J Health Econ Outcomes Res. 2021;8(2):35–44. doi:10.36469/001c.26129
   Google Scholar
• Sim JJ, Batech M, Hever A, et al. Distribution of biopsy-proven presumed primary glomerulonephropathies in 2000–2011 among a racially and ethnically diverse US population. Am J Kidney Dis. 2016;68(4):533–544. doi:10.1053/j.ajkd.2016.03.416
   PubMed Web of Science ®Google Scholar
• Moldoveanu Z, Wyatt RJ, Lee JY, et al. Patients with IgA nephropathy have increased serum galactose-deficient IgA1 levels. Kidney Int. 2007;71(11):1148–1154. doi:10.1038/sj.ki.5002185
   PubMed Web of Science ®Google Scholar
• Floege J. Fatal attraction: immunoglobulin A and the glomerular mesangium. J Am Soc Nephrol. 2019;30(7):1139–1141. doi:10.1681/ASN.2019040373
   PubMed Web of Science ®Google Scholar
• Canetta PA, Kiryluk K, Appel GB. Glomerular diseases: emerging tests and therapies for IgA nephropathy. CJASN. 2014;9(3):617–625. doi:10.2215/CJN.07260713
   PubMed Web of Science ®Google Scholar
• Kiryluk K, Novak J. The genetics and immunobiology of IgA nephropathy. J Clin Invest. 2014;124(6):2325–2332. doi:10.1172/jci74475
   PubMed Web of Science ®Google Scholar
• Reid S, Cawthon PM, Craig JC, Samuels JA, Molony DA, Strippoli GF. Non-immunosuppressive treatment for IgA nephropathy. Cochrane Database Syst Rev. 2011. doi:10.1002/14651858.CD003962.pub2
   Web of Science ®Google Scholar
• Barratt J, Rovin BH, Cattran D, Trimarchi H, Zhang H. Why target the gut to treat IgA nephropathy? KI Reports. 2020;5(10):1620–1624. doi:10.1016/j.ekir.2020.08.009
   PubMed Web of Science ®Google Scholar
• Yeo SC, Goh SM, Barratt J. Is immunoglobulin A nephropathy different in different ethnic populations? Nephrology. 2019;24(9):885–895. doi:10.1111/nep.13592
   PubMed Web of Science ®Google Scholar
• Duan ZY, Cai GY, Chen YZ, et al. Aging promotes progression of IgA nephropathy: a systematic review and meta-analysis. Am J Nephrol. 2013;38(3):241–252. doi:10.1159/000354646
   PubMed Web of Science ®Google Scholar
• Schena FP. A retrospective analysis of the natural history of primary IgA nephropathy worldwide. Am J Med. 1990;89(2):209–215. doi:10.1016/0002-9343(90)90300-3
   PubMed Web of Science ®Google Scholar
• Knoop T, Vikse BE, Svarstad E, Leh S, Reisæter AV, Bjørneklett R. Mortality in patients with IgA nephropathy. Am J Kidney Dis. 2013;62(5):883–890. doi:10.1053/j.ajkd.2013.04.019
   PubMed Web of Science ®Google Scholar
• Hastings MC, Bursac Z, Julian BA, et al. Life expectancy for patients from the Southeastern United States With IgA nephropathy. Kidney Int Rep. 2018;3(1):99–104. doi:10.1016/j.ekir.2017.08.008
   PubMed Web of Science ®Google Scholar
• Arroyo AH, Bomback AS, Butler B, et al. Predictors of outcome for severe IgA nephropathy in a multi-ethnic U.S. cohort. CN. 2015;84:145–155. doi:10.5414/CN108556
   Google Scholar
• Rauen T, Wied S, Fitzner C, et al. After ten years of follow-up, no difference between supportive care plus immunosuppression and supportive care alone in IgA nephropathy. Kidney Int. 2020;98(4):1044–1052. doi:10.1016/j.kint.2020.04.046
   PubMed Web of Science ®Google Scholar
• Hiragi S, Tamura H, Goto R, Kuroda T. The effect of model selection on cost-effectiveness research: a comparison of kidney function-based microsimulation and disease grade-based microsimulation in chronic kidney disease modeling. BMC Med Inform Decis Mak. 2018;18(1):94. doi:10.1186/s12911-018-0678-7
   PubMedGoogle Scholar
• Libório AB, Santos JPL, Minete NFA, et al. Proteinuria is associated with quality of life and depression in adults with primary glomerulopathy and preserved renal function. PLoS One. 2012;7(5):e37763. doi:10.1371/journal.pone.0037763
   PubMed Web of Science ®Google Scholar
• Infante B, Rossini M, Di Lorenzo A, et al. Recurrence of immunoglobulin A nephropathy after kidney transplantation: a narrative review of the incidence, risk factors, pathophysiology and management of immunosuppressive therapy. Clin Kidney J. 2020;13(5):758–767. doi:10.1093/ckj/sfaa060
   PubMed Web of Science ®Google Scholar
• Allen PJ, Chadban SJ, Craig JC, et al. Recurrent glomerulonephritis after kidney transplantation: risk factors and allograft outcomes. Kidney Int. 2017;92(2):461–469. doi:10.1016/j.kint.2017.03.015
   PubMed Web of Science ®Google Scholar
• Ponticelli C, Glassock RJ. Posttransplant recurrence of primary glomerulonephritis. Clin J Am Soc Nephrol. 2010;5(12):2363–2372. doi:10.2215/CJN.06720810
   PubMed Web of Science ®Google Scholar
• Ponticelli C, Traversi L, Banfi G. Renal transplantation in patients with IgA mesangial glomerulonephritis. Pediatr Transplant. 2004;8(4):334–338. doi:10.1111/j.1399-3046.2004.00177.x
   PubMed Web of Science ®Google Scholar
• Rovin BH, Adler SG, Barratt J, et al. KDIGO 2021 clinical practice guideline for the management of glomerular diseases. Kidney Int. 2021;100(4):S1–S276. doi:10.1016/j.kint.2021.05.021
   PubMed Web of Science ®Google Scholar
• Fellstrom BC, Barratt J, Cook H, et al. Targeted-release budesonide versus placebo in patients with IgA nephropathy (NEFIGAN): a double-blind, randomised, placebo-controlled phase 2b trial. Lancet. 2017;389(10084):2117–2127. doi:10.1016/S0140-6736(17)30550-0
   PubMed Web of Science ®Google Scholar
• Watts P, Smith A. TARGIT technology: coated starch capsules for site-specific drug delivery into the lower gastrointestinal tract. Expert Opin Drug Deliv. 2005;2(1):159–167. doi:10.1517/17425247.2.1.159
   PubMedGoogle Scholar
• Calliditas Therapeutics AB. TARPEYO (Budesonide) US prescribing information; 2021. Available from: https://www.accessdata.fda.gov/drugsatfda_docs/label/2021/215935s000lbl.pdf. Accessed March 16, 2023.
   Google Scholar
• Barratt J, Lafayette R, Kristensen J, et al. Results from part A of the multi-center, double-blind, randomized, placebo-controlled NefIgArd trial evaluated targeted-release formulation of budesonide for the treatment of primary immunoglobulin A nephropathy. Kidney Int. 2023;103. doi:10.1016/j.kint.2022.09.017
   Web of Science ®Google Scholar
• ICER’s Reference Case for Economic Evaluations. Principles and rationale. Institute for Clinical and Economic Review; 2020. https://icer.org/wp-content/uploads/2020/10/ICER_Reference_Case_013120.pdf. Accessed June 25, 2021.
   Google Scholar
• Calliditas Therapeutics AB. Data on file: clinical study report – a randomized, double-blind, placebo-controlled study to evaluate efficacy and safety of nefecon in patients with primary IgA nephropathy at risk of progressing to end-stage renal disease (NefIgArd) (data cutoff date of 05 October 2020 for part a analysis) – version 1.0; 2021.
   Google Scholar
• Kent S, Schlackow I, Lozano-Kühne J, et al. What is the impact of chronic kidney disease stage and cardiovascular disease on the annual cost of hospital care in moderate-to-severe kidney disease? BMC Nephrol. 2015;16(1):65. doi:10.1186/s12882-015-0054-0
   PubMedGoogle Scholar
• R Core Team. R: a language and environment for statistical computing; 2013. Available from: http://www.R-project.org/. Accessed March 16, 2023.
   Google Scholar
• Ortiz F, Harjutsalo V, Helanterä I, Lempinen M, Forsblom C, Groop PH. Long-term mortality after kidney transplantation in a nationwide cohort of patients with type 1 diabetes in Finland. Diabetes Care. 2019;42(1):55–61. doi:10.2337/dc18-1029
   PubMed Web of Science ®Google Scholar
• CPI inflation calculator; 2021. Available from: https://www.bls.gov/data/inflation_calculator.htm. Accessed June 25, 2021.
   Google Scholar
• Calliditas Therapeutics AB. Data on file: price of TARPEYO©; 2021.
   Google Scholar
• HCUPnet. HCUPnet; 2015. Available from: https://hcupnet.ahrq.gov/. Accessed April 15, 2021.
   Google Scholar
• Statistics The Kidney Project. The kidney project; 2021. Available from: https://pharm.ucsf.edu/kidney/need/statistics. Accessed June 25, 2021.
   Google Scholar
• Annual Data Report. USRDS; 2021. Available from: https://adr.usrds.org/2020/end-stage-renal-disease/9-healthcare-expenditures-for-persons-with-esrd. Accessed June 25, 2021.
   Google Scholar
• OPTUM. Kidney resource services – network access; 2017. Available from: https://www.optum.com/content/dam/optum3/optum/en/resources/white-papers/kidney-resources-services-network-access.pdf. Accessed March 16, 2023.
   Google Scholar
• Bentley TS, Ortner NJ. Milliman research report: 2020 U.S. Organ and Tissue Transplants: Cost estimates, discussion, and emerging issues. Available from: https://www.milliman.com/-/media/milliman/pdfs/articles/2020-us-organ-tissue-transplants.ashx. Accessed March 16, 2023.
   Google Scholar
• Kerr M, Matthews B, Medcalf JF. End-of-life care for people with chronic kidney disease: cause of death, place of death and hospital costs. Nephrology Dialysis Transplantation | Oxford Academic; 2017. Available from: https://academic.oup.com/ndt/article/32/9/1504/3059442. Accessed March 16, 2023.
   Google Scholar
• Historical rates tables - USD | xe; 2021. Available from: https://www.xe.com/currencytables/?from=USD. Accessed June 25, 2021.
   Google Scholar
• Bekelman JE, Halpern SD, Blankart CR, et al. Comparison of site of death, health care utilization, and hospital expenditures for patients dying with cancer in 7 developed Countries. JAMA. 2016;315(3):272–283. doi:10.1001/jama.2015.18603
   PubMed Web of Science ®Google Scholar
• van Haalen H, Jackson J, Spinowitz B, Milligan G, Moon R. Impact of chronic kidney disease and anemia on health-related quality of life and work productivity: analysis of multinational real-world data. BMC Nephrol. 2020;21(1):88. doi:10.1186/s12882-020-01746-4
   PubMed Web of Science ®Google Scholar
• Statista. U.S. employment rate by age 2020; 2021. Available from: https://www.statista.com/statistics/217899/us-employment-rate-by-age/. Accessed June 25, 2021.
   Google Scholar
• Bureau of Labor Statistics. Usual weekly earnings of wage and salary workers first quarter; 2021. Available from: https://www.bls.gov/news.release/pdf/wkyeng.pdf. Accessed November 16, 2021.
   Google Scholar
• Gorodetskaya I, Zenios S, Mcculloch CE, et al. Health-related quality of life and estimates of utility in chronic kidney disease. Kidney Int. 2005;68(6):2801–2808. doi:10.1111/j.1523-1755.2005.00752.x
   PubMed Web of Science ®Google Scholar
• Thompson A, Carroll K, Inker L, et al. Proteinuria reduction as a surrogate end point in trials of IgA nephropathy. Clin J Am Soc Nephrol. 2019;14(3):469–481. doi:10.2215/CJN.08600718
   PubMed Web of Science ®Google Scholar
• Mustafa R, Bloudek L, Fox G, et al. Treatments for anemia in chronic kidney disease: effectiveness and value; evidence report. Institute Clin Eco Rev. 2021;9:5.
   Google Scholar
• Lin E, Chertow GM, Yan B, Malcolm E, Goldhaber-Fiebert JD. Cost-effectiveness of multidisciplinary care in mild to moderate chronic kidney disease in the United States: a modeling study. PLoS Med. 2018;15(3):e1002532. doi:10.1371/journal.pmed.1002532
   PubMed Web of Science ®Google Scholar
• Sugrue DM, Ward T, Rai S, McEwan P, van Haalen HGM. Economic modelling of chronic kidney disease: a systematic literature review to inform conceptual model design. Pharmaco Eco. 2019;37(12):1451–1468. doi:10.1007/s40273-019-00835-z
   PubMed Web of Science ®Google Scholar
• Tonelli M, Klarenbach S, Wiebe N, Shrive F, Hemmelgarn B, Manns B. Stimulating agents for anemia of chronic kidney disease: systematic review and economic evaluation [technology report number 106]. Canadian Agency Drugs Technol Health. 2008;2008:15.
   Google Scholar
• National Guideline Centre. RRT and conservative management. Cost-effectiveness analysis: HDF versus high flux HD. NICE. 2018;2008:105.
   Google Scholar
• Tice J, Mandrik O, Thokala P, et al. Voclosporin and belimumab for lupus nephritis: effectiveness and value; evidence report. Institute Clin Eco Rev. 2021;20:1105.
   Google Scholar
• Bhachu JS, Scionti K, Muto M, Molyneux K, Barratt J. Targeted release-budesonide (NEFECON) modifies circulating IgA-IgG immune complex levels and levels of poorly O-Galactosylated IgA in IgAN. Kidney Dis. 2018;4:121–122.
   Google Scholar
• Thompson A, Carroll K, Inker LA, et al. Proteinuria reduction as a surrogate end point in trials of IgA nephropathy. CJASN. 2019;14(3):469–481. doi:10.2215/cjn.08600718
   PubMed Web of Science ®Google Scholar