title: Cost-minimization analysis of IgPro20, a subcutaneous immunoglobulin, in Japanese patients with primary immunodeficiency.
abstract: OBJECTIVE
  IgPro20, Hizentra(®) an L-proline-stabilized 20% human subcutaneous immunoglobulin (SCIG), has been shown in a Phase III pivotal study to be well tolerated and efficacious in adult and pediatric Japanese patients with primary immunodeficiency. Economic aspects of SCIG treatment in comparison with previous intravenous immunoglobulin (IVIG) therapy were analyzed in this Phase III study in Japan.
  
  
  METHODS
  Twenty-four Japanese patients with primary immunodeficiency on IVIG treatment were switched to IgPro20 at an equivalent dose (full analysis set). The study consisted of a screening period, an IVIG treatment period with 3 planned infusions every 3 or 4 weeks, a 12-week SCIG wash-in and wash-out period, and a 12-week SCIG efficacy period. The difference in medical cost and productivity loss resulting from changes in hospital frequency between the SCIG and IVIG treatment was evaluated. Information about treatment cost was collected as part of the Life Quality Index questionnaire. In addition, productivity loss and hospital-related absenteeism were evaluated.
  
  
  RESULTS
  Life Quality Index scores for all domains were higher with SCIG than with IVIG in this patient population. In the full analysis set, the mean (SD) Life Quality Index score of the Costs domain increased from 45.1 (26.34) at Week 1 (IVIG period) to 71.9 (18.52) at Week 24 (end of the SCIG efficacy period), representing a mean change of 26.74 and a large score improvement effect size (1.01). Median productivity loss was reduced by 60% from baseline to Weeks 12 and 24. This resulted in a reduction in costs of JPY 10,875 per patient per month at Weeks 12 and 24. Subcutaneous treatment with IgPro20 also reduced hospital-related absenteeism. The number of patients, parents, or guardians who were not absent from work or housework duties and had no reduction in working time increased from 4 (17.4%) at Week 1 to 9 (39.1%) at Week 24. Similar results were obtained in the per-protocol set (n = 21).
  
  
  CONCLUSIONS
  Switching from IVIG to SCIG reduced markedly productivity loss and hospital-related absenteeism. The reduction in hospital visit frequency due to the use of home-based IgG therapy enabled by the change in administration route is expected to produce an important pharmacoeconomic benefit in Japan. Study Code: ZLB06_002CR, ClinicalTrials.gov identifier: NCT01199705.
authors.affiliation: Department of Drug Policy and Management, Graduate School of Pharmaceutical Sciences, University of Tokyo, Japan. Electronic address: atarui1@mac.com.
authors.forename: Ataru
authors.initials: A
authors.lastname: Igarashi
authors.affiliation: Department of Pediatrics, Graduate School of Medicine and Pharmaceutical Sciences, University of Toyama, Japan.
authors.forename: Hirokazu
authors.initials: H
authors.lastname: Kanegane
authors.affiliation: CSL Behring K.K., Tokyo, Japan.
authors.forename: Midori
authors.initials: M
authors.lastname: Kobayashi
authors.affiliation: Department of Pediatrics, Graduate School of Medicine and Pharmaceutical Sciences, University of Toyama, Japan.
authors.forename: Toshio
authors.initials: T
authors.lastname: Miyawaki
authors.affiliation: Department of Drug Policy and Management, Graduate School of Pharmaceutical Sciences, University of Tokyo, Japan.
authors.forename: Kiichiro
authors.initials: K
authors.lastname: Tsutani
mesh_terms: D000041:Absenteeism; D000293:Adolescent; D000328:Adult; D002648:Child; D002675:Child, Preschool; D017046:Cost Savings; D003365:Costs and Cost Analysis; D005260:Female; D006760:Hospitalization / Q000191:economics / Q000706:statistics & numerical data; D006801:Humans; D007074:Immunoglobulin G / Q000191:economics* / Q000627:therapeutic use; D016756:Immunoglobulins, Intravenous; D007153:Immunologic Deficiency Syndromes / Q000188:drug therapy*; D055104:Infusions, Subcutaneous; D007564:Japan; D008297:Male; D008875:Middle Aged; D012646:Self Administration; D055815:Young Adult
chemical_list: C558471:Hizentra; D007074:Immunoglobulin G; D016756:Immunoglobulins, Intravenous
keywords: cost minimization; home-based therapy; immunoglobulin replacement therapy; pharmacoeconomics; productivity loss; subcutaneous IgG