Quantifying the economic effects of ravulizumab versus eculizumab treatment in patients with atypical hemolytic uremic syndrome

References

• Junkins CC, Kent E, Litzelman K, et al. Cancer across the ages: a narrative review of caregiver burden for patients of all ages. J Psychosoc Oncol. 2020;38(6):782–798.
   PubMed Web of Science ®Google Scholar
• Yu H, Wang X, He R, et al. Measuring the caregiver burden of caring for community-residing people with Alzheimer's disease. PLOS One. 2015;10(7):e0132168.
   PubMed Web of Science ®Google Scholar
• Wimo A, Reed CC, Dodel R, et al. The GERAS study: a prospective observational study of costs and resource use in community dwellers with Alzheimer's disease in three European countries – study design and baseline findings. JAD. 2013;36(2):385–399.
   Google Scholar
• White W. A rare disease patient/caregiver perspective on fair pricing and access to gene-based therapies. Gene Ther. 2019;27(10–11):474–481.
   PubMedGoogle Scholar
• Linertová R, García-Pérez L, Gorostiza I. Cost-of-illness in rare diseases. Adv Exp Med Biol. 2017;1031:283–297.
   PubMed Web of Science ®Google Scholar
• Campistol JM, Arias M, Ariceta G, et al. An update for atypical haemolytic uraemic syndrome: diagnosis and treatment. A consensus document. Nefrologia. 2015;35(5):421–447.
   PubMed Web of Science ®Google Scholar
• Fakhouri F, Zuber J, Frémeaux-Bacchi V, et al. Haemolytic uraemic syndrome. Lancet. 2017;390(10095):681–696.
   PubMed Web of Science ®Google Scholar
• George JN, Nester CM. Syndromes of thrombotic microangiopathy. N Engl J Med. 2014;371(7):654–666.
   PubMed Web of Science ®Google Scholar
• Sellier-Leclerc AL, Fremeaux-Bacchi V, Dragon-Durey MA, et al. Differential impact of complement mutations on clinical characteristics in atypical hemolytic uremic syndrome. J Am Soc Nephrol. 2007;18(8):2392–2400.
   PubMed Web of Science ®Google Scholar
• Fremeaux-Bacchi V, Fakhouri F, Garnier A, et al. Genetics and outcome of atypical hemolytic uremic syndrome: a Nationwide French Series Comparing Children and Adults. CJASN. 2013;8(4):554–562.
   PubMed Web of Science ®Google Scholar
• US Food and Drug Administration. Eculizumab (SOLIRIS) Prescribing Information, 2020. [cited June 2021]. https://www.accessdata.fda.gov/drugsatfda_docs/label/2020/125166s434lbl.pdf
   Google Scholar
• European Medicines Agency. Eculizumab (SOLIRIS). 2020. [cited June 2021]. https://www.ema.europa.eu/en/documents/product-information/soliris-epar-product-information_en.pdf
   Google Scholar
• European Medicines Agency. Ravulizumab (ULTOMIRIS). [cited June 2021]. 2021. https://www.ema.europa.eu/en/documents/product-information/ultomiris-epar-product-information_en.pdf
   Google Scholar
• US Food and Drug Administration. Ravulizumab (ULTOMIRIS) Prescribing Information, 2021. [cited June 2021]. https://www.accessdata.fda.gov/drugsatfda_docs/label/2021/761108s012lbl.pdf
   Google Scholar
• Legendre CM, Licht C, Muus P, et al. Terminal complement inhibitor eculizumab in atypical hemolytic–uremic syndrome. N Engl J Med. 2013;368(23):2169–2181.
   PubMed Web of Science ®Google Scholar
• Zuber J, Frimat M, Caillard S, et al. Use of highly individualized complement blockade has revolutionized clinical outcomes after kidney transplantation and renal epidemiology of atypical hemolytic uremic syndrome. J Am Soc Nephrol. 2019;30(12):2449–2463.
   PubMed Web of Science ®Google Scholar
• Fakhouri F, Hourmant M, Campistol JM, et al. Terminal complement inhibitor eculizumab in adult patients with atypical hemolytic uremic syndrome: a single-arm, open-label trial. Am J Kidney Dis. 2016;68(1):84–93.
   PubMed Web of Science ®Google Scholar
• Greenbaum LA, Fila M, Ardissino G, et al. Eculizumab is a safe and effective treatment in pediatric patients with atypical hemolytic uremic syndrome. Kidney Int. 2016;89(3):701–711.
   PubMed Web of Science ®Google Scholar
• Rondeau E, Cataland SR, Al-Dakkak I, et al. Eculizumab safety: five-year experience from the global atypical hemolytic uremic syndrome registry. Kidney Int Rep. 2019;4(11):1568–1576.
   PubMed Web of Science ®Google Scholar
• Licht C, Greenbaum LA, Muus P, et al. Efficacy and safety of eculizumab in atypical hemolytic uremic syndrome from 2-year extensions of phase 2 studies. Kidney Int. 2015;87(5):1061–1073.
   PubMed Web of Science ®Google Scholar
• Ariceta G. Optimal duration of treatment with eculizumab in atypical hemolytic uremic syndrome (aHUS)-a question to be addressed in a scientific way. Pediatr Nephrol. 2019;34(5):943–949.
   PubMed Web of Science ®Google Scholar
• Yüksel S, Evrengül H, Özçakar ZB, et al. First-line, early and long-term eculizumab therapy in atypical hemolytic uremic syndrome: a case series in pediatric patients. Paediatr Drugs. 2016;18(6):413–420.
   PubMed Web of Science ®Google Scholar
• Rondeau E, Scully M, Ariceta G, et al. The long-acting C5 inhibitor, ravulizumab, is effective and safe in adult patients with atypical hemolytic uremic syndrome naive to complement inhibitor treatment. Kidney Int. 2020;97(6):1287–1296.
   PubMed Web of Science ®Google Scholar
• Barbour T, Scully M, Ariceta G, et al. Long-term efficacy and safety of the long-acting complement C5 inhibitor ravulizumab for the treatment of atypical hemolytic uremic syndrome in adults. Kidney Int Rep. 2021;6(6):1603–1613.
   PubMed Web of Science ®Google Scholar
• Tanaka K, Adams B, Aris AM, et al. The long-acting C5 inhibitor, ravulizumab, is efficacious and safe in pediatric patients with atypical hemolytic uremic syndrome previously treated with eculizumab. Pediatr Nephrol. 2021;36(4):889–898.
   PubMed Web of Science ®Google Scholar
• Ariceta G, Dixon BP, Kim SH, et al. The long-acting C5 inhibitor, ravulizumab, is effective and safe in pediatric patients with atypical hemolytic uremic syndrome naïve to complement inhibitor treatment. Kidney Int. 2021;100(1):225–237.
   PubMed Web of Science ®Google Scholar
• Wang Y, Johnston K, Popoff E, et al. A US cost-minimization model comparing ravulizumab versus eculizumab for the treatment of atypical hemolytic uremic syndrome. J Med Econ. 2020;23(12):1503–1515.
   PubMed Web of Science ®Google Scholar
• Verbooy K, Hoefman R, van Exel J, et al. Time is money: investigating the value of leisure time and unpaid work. Value Health. 2018;21(12):1428–1436.
   PubMed Web of Science ®Google Scholar
• Russell LB. Completing costs: patients' time. Med Care. 2009;47(7 Suppl 1):S89–S93.
   PubMed Web of Science ®Google Scholar
• Williams K, Aggio D, Chen P, et al. Utility values associated with atypical hemolytic uremic syndrome-related attributes: a discrete choice experiment in five countries. Pharmacoeconomics. 2021;39(8):901–912.
   PubMed Web of Science ®Google Scholar
• Brouwer WB, Koopmanschap MA, Rutten FF. Productivity costs in cost-effectiveness analysis: numerator or denominator: a further discussion. Health Econ. 1997;6(5):511–514.
   PubMed Web of Science ®Google Scholar
• van den Hout WB. The value of productivity: human-capital versus friction-cost method. Ann Rheum Dis. 2010;69(Suppl 1):i89–91.
   PubMed Web of Science ®Google Scholar
• Abrams HR, Leeds HS, Russell HV, et al. Factors influencing family burden in pediatric hematology/oncology encounters. J Patient Cent Res Rev. 2019;6(4):243–251.
   PubMed Web of Science ®Google Scholar
• Tai B-WB, Bae YH, Le QA. A systematic review of health economic evaluation studies using the patient's perspective. Value Health. 2016;19(6):903–908.
   PubMed Web of Science ®Google Scholar
• de Meijer C, Brouwer W, Koopmanschap M, et al. The value of informal care-a further investigation of the feasibility of contingent valuation in informal caregivers. Health Econ. 2010;19(7):755–771.
   PubMed Web of Science ®Google Scholar
• van Exel NJ, Brouwer WB, van den Berg B, et al. What really matters: an inquiry into the relative importance of dimensions of informal caregiver burden. Clin Rehabil. 2004;18(6):683–693.
   PubMed Web of Science ®Google Scholar
• Heffernan M, Meit M, Powers M, et al. Business leaders' attitudes about value of employee and community health. J Public Health Manag Pract. 2020;26(5):493–496.
   PubMed Web of Science ®Google Scholar
• Sanders GD, Neumann PJ, Basu A, et al. Recommendations for conduct, methodological practices, and reporting of cost-effectiveness analyses: second panel on cost-effectiveness in health and medicine. JAMA. 2016;316(10):1093–1103.
   PubMed Web of Science ®Google Scholar
• Grosse SD, Pike J, Soelaeman R, et al. Quantifying family spillover effects in economic evaluations: measurement and valuation of informal care time. Pharmacoeconomics. 2019;37(4):461–473.
   PubMed Web of Science ®Google Scholar
• Basu A, Meltzer D. Implications of spillover effects within the family for medical cost-effectiveness analysis. J Health Econ. 2005;24(4):751–773.
   PubMed Web of Science ®Google Scholar
• Neri L, Lucidi V, Catastini P, et al. Caregiver burden and vocational participation among parents of adolescents with CF. Pediatr Pulmonol. 2016;51(3):243–252.
   PubMed Web of Science ®Google Scholar
• Bernuy-Guevara C, Chehade H, Muller YD, et al. The inhibition of complement system in formal and emerging indications: results from parallel one-stage pairwise and network meta-analyses of clinical trials and real-life data studies. Biomedicines. 2020;8(9):355.
   PubMed Web of Science ®Google Scholar
• Raina R, Grewal MK, Radhakrishnan Y, et al. Optimal management of atypical hemolytic uremic disease: challenges and solutions. Int J Nephrol Renovasc Dis. 2019;12:183–204.
   PubMed Web of Science ®Google Scholar
• Menne J, Delmas Y, Fakhouri F, et al. Outcomes in patients with atypical hemolytic uremic syndrome treated with eculizumab in a long-term observational study. BMC Nephrol. 2019;20(1):125.
   PubMedGoogle Scholar
• Tomazos IH, Cataland S, Chen P, et al. Comparative efficacy of ravulizumab and eculizumab in the treatment of atypical hemolytic uremic syndrome: an indirect comparison using clinical trial data. Clin Nephrol. 2021. DOI:10.5414/CN110516.
   Google Scholar