title: Cost-effectiveness of adding carfilzomib to lenalidomide and dexamethasone in relapsed multiple myeloma from a US perspective.
abstract: OBJECTIVE
  To assess the economic value of carfilzomib (Kyprolis), this study developed the Kyprolis Global Economic Model (K-GEM), which examined from a United States (US) payer perspective the cost-effectiveness of carfilzomib-lenalidomide-dexamethasone (KRd) versus lenalidomide-dexamethasone (Rd) in relapsed multiple myeloma (RMM; 1-3 prior therapies) based on results from the phase III ASPIRE trial that directly compared these regimens.
  
  
  METHODS
  A partitioned survival model that included three health states of progression-free (on or off treatment), post-progression, and death was developed. Using ASPIRE data, the effect of treatment regimens as administered in the trial was assessed for progression-free survival and overall survival (OS). Treatment effects were estimated with parametric regression models adjusting for baseline patient characteristics and applied over a lifetime horizon. US Surveillance, Epidemiology and End Results (1984-2014) registry data were matched to ASPIRE patients to extrapolate OS beyond the trial. Estimated survival was adjusted to account for utilities across health states. The K-GEM considered the total direct costs (pharmacy/medical) of care for patients treated with KRd and Rd.
  
  
  RESULTS
  KRd was estimated to be more effective compared to Rd, providing 1.99 life year and 1.67 quality-adjusted life year (QALY) gains over the modeled horizon. KRd-treated patients incurred $179,393 in total additional costs. The incremental cost-effectiveness ratio (ICER) was $107,520 per QALY.
  
  
  CONCLUSIONS
  Extrapolated survival functions present the greatest uncertainty in the modeled results. Utilities were derived from a combination of sources and assumed to reflect how US patients value their health state.
  
  
  CONCLUSIONS
  The K-GEM showed KRd is cost-effective, with an ICER of $107,520 per QALY gained against Rd for the treatment of patients with RMM (1-3 prior therapies) at a willingness-to-pay threshold of $150,000. Reimbursement of KRd for patients with RMM may represent an efficient allocation of the healthcare budget.
authors.affiliation: a Myeloma Program , University of Chicago, Chicago , IL , USA.
authors.forename: Andrzej J
authors.initials: AJ
authors.lastname: Jakubowiak
authors.affiliation: b Global Health Economics , Amgen (Europe) GmbH, Zug , Switzerland.
authors.forename: Marco
authors.initials: M
authors.lastname: Campioni
authors.affiliation: c Modeling and Simulation , Evidera, Budapest , Hungary.
authors.forename: Ágnes
authors.initials: Á
authors.lastname: Benedict
authors.affiliation: c Modeling and Simulation , Evidera, Budapest , Hungary.
authors.forename: Ivan
authors.initials: I
authors.lastname: Houisse
authors.affiliation: c Modeling and Simulation , Evidera, Budapest , Hungary.
authors.forename: Eszter
authors.initials: E
authors.lastname: Tichy
authors.affiliation: b Global Health Economics , Amgen (Europe) GmbH, Zug , Switzerland.
authors.forename: Andromachi
authors.initials: A
authors.lastname: Giannopoulou
authors.affiliation: d Global Development , Amgen Inc., Thousand Oaks , CA , USA.
authors.forename: Sanjay K
authors.initials: SK
authors.lastname: Aggarwal
authors.affiliation: d Global Development , Amgen Inc., Thousand Oaks , CA , USA.
authors.forename: Beth L
authors.initials: BL
authors.lastname: Barber
authors.affiliation: e Global Health Economics , Amgen Inc. San Francisco , CA , USA.
authors.forename: Sumeet
authors.initials: S
authors.lastname: Panjabi
mesh_terms: D000971:Antineoplastic Combined Chemotherapy Protocols / Q000191:economics*; D003362:Cost-Benefit Analysis; D003907:Dexamethasone / Q000191:economics* / Q000627:therapeutic use*; D018572:Disease-Free Survival; D006801:Humans; D000077269:Lenalidomide; D018803:Models, Economic; D009101:Multiple Myeloma / Q000188:drug therapy*; D009842:Oligopeptides / Q000191:economics* / Q000627:therapeutic use*; D011788:Quality of Life; D019057:Quality-Adjusted Life Years; D012008:Recurrence; D013792:Thalidomide / Q000031:analogs & derivatives* / Q000191:economics / Q000627:therapeutic use; D014481:United States
chemical_list: D009842:Oligopeptides; D013792:Thalidomide; C524865:carfilzomib; D003907:Dexamethasone; D000077269:Lenalidomide
keywords: Carfilzomib; Cost-effectiveness; Dexamethasone; Lenalidomide; Multiple myeloma; Relapsed