title: Cost-Effectiveness Analysis of Regorafenib for Gastrointestinal Stromal Tumour (GIST) in Germany.
abstract: BACKGROUND
  No study has compared the cost-effectiveness of active treatment options for unresectable or metastatic gastrointestinal stromal tumours in patients who progressed on or are intolerant to prior treatment with imatinib and sunitinib. The aim of this study was to estimate the cost-effectiveness of regorafenib compared to imatinib rechallenge in this setting in Germany.
  
  
  METHODS
  Hazard ratios for progression-free (PFS) and overall survival (OS) with regorafenib versus imatinib rechallenge were estimated by indirect comparison. A state distribution model was used to simulate progression, mortality and treatment costs over a lifetime horizon. Drug acquisition costs and utilities were derived from clinical trial data and published literature; non-drug costs were not included. The outcomes measured were treatment costs, life-years (LYs) and quality-adjusted life-years (QALYs).
  
  
  RESULTS
  The indirect comparison suggested that median PFS and OS were longer with regorafenib compared to imatinib but results were not statistically significant. Regorafenib versus imatinib rechallenge was estimated to have hazard ratios of 0.58 (95% CI 0.31-1.11) for PFS and 0.77 (95% CI 0.34-1.77) for OS, with substantial uncertainty due to the rarity of the disease and small number of patients within the trials. Regorafenib treatment per patient over a lifetime horizon provided an additional 0.61 LYs and 0.42 QALYs over imatinib rechallenge, with additional direct drug costs of €8,773. The incremental cost-effectiveness ratio was €21,127 per QALY gained. At a cost-effectiveness threshold of €50,000 per QALY, regorafenib had a 67% probability of being cost-effective.
  
  
  CONCLUSIONS
  Based on the currently available clinical data, this analysis suggests that regorafenib is cost-effective compared with imatinib rechallenge in Germany.
authors.affiliation: Bayer HealthCare Germany, Leverkusen, Germany.
authors.forename: David
authors.initials: D
authors.lastname: Tamoschus
authors.affiliation: Bayer HealthCare Germany, Leverkusen, Germany.
authors.forename: Katja
authors.initials: K
authors.lastname: Draexler
authors.affiliation: Bayer HealthCare Pharmaceuticals, Whippany, NJ, USA.
authors.forename: Jane
authors.initials: J
authors.lastname: Chang
authors.affiliation: Bayer HealthCare Pharmaceuticals, Whippany, NJ, USA.
authors.forename: Christopher
authors.initials: C
authors.lastname: Ngai
authors.affiliation: QuintilesIMS, 210 Pentonville Road, Kings Cross, London, N1 9JY, UK.
authors.forename: Matthew
authors.initials: M
authors.lastname: Madin-Warburton
authors.affiliation: QuintilesIMS, 210 Pentonville Road, Kings Cross, London, N1 9JY, UK. ashley.pitcher@quintilesims.com.
authors.forename: Ashley
authors.initials: A
authors.lastname: Pitcher
mesh_terms: D000368:Aged; D000970:Antineoplastic Agents / Q000627:therapeutic use*; D003362:Cost-Benefit Analysis; D016527:Drug Costs; D005260:Female; D005770:Gastrointestinal Neoplasms / Q000188:drug therapy* / Q000401:mortality; D046152:Gastrointestinal Stromal Tumors / Q000188:drug therapy* / Q000401:mortality; D006801:Humans; D000068877:Imatinib Mesylate / Q000627:therapeutic use; D008297:Male; D008875:Middle Aged; D010671:Phenylurea Compounds / Q000627:therapeutic use*; D011725:Pyridines / Q000627:therapeutic use*; D019057:Quality-Adjusted Life Years
chemical_list: D000970:Antineoplastic Agents; D010671:Phenylurea Compounds; D011725:Pyridines; C559147:regorafenib; D000068877:Imatinib Mesylate