title: Orphan drugs revisited: cost-effectiveness analysis of the addition of mifamurtide to the conventional treatment of osteosarcoma.
abstract: BACKGROUND
  Mepact(®) (mifamurtide) is the first drug approved for the treatment of high-grade resectable non-metastatic osteosarcoma in patients aged 2-30 in the last 20 years. It follows a randomized clinical trial showing a statistically-significant and clinically-relevant decrease in the risk of death without compromising safety.
  
  
  OBJECTIVE
  This study assessed the cost-effectiveness and budget impact of mifamurtide.
  
  
  METHODS
  The economic evaluation was done on a hypothetical cohort of young patients under the age of 30 with high-grade, non-metastatic, resectable osteosarcoma. Standard chemotherapy without mifamurtide was used as comparator. A Markov model was adapted using Spanish costs and the results of the INT-0133 Phase III study. The analysis has been carried out from the perspective of the Spanish National Health Service, with a time horizon of up to 60 years in the base analysis.
  
  
  RESULTS
  The observed greater efficacy of mifamurtide in the trial translates into a gain of 3.03 (undiscounted) and 1.33 (discounted) quality-adjusted life years at an additional cost of €102,000. The estimated budgetary impact of using mifamurtide in 10-100% of the potential population would cost €671,000 and €6.7 million respectively.
  
  
  CONCLUSIONS
  The cost per quality-adjusted life years gained with mifamurtide in Spain is in the low band (<€100,000) of the iCERs obtained by other orphan drugs and would have a limited and affordable cost in Spain.
authors.affiliation: Oblikue Consulting S.L., C/Josep Irla i Bosh 5-7, 08034 Barcelona, Spain.
authors.forename: Max
authors.initials: M
authors.lastname: Brosa
authors.forename: Xavier
authors.initials: X
authors.lastname: García del Muro
authors.forename: Jaume
authors.initials: J
authors.lastname: Mora
authors.forename: Alba
authors.initials: A
authors.lastname: Villacampa
authors.forename: Tamara
authors.initials: T
authors.lastname: Pozo-Rubio
authors.forename: Laia
authors.initials: L
authors.lastname: Cubells
authors.forename: Carmen
authors.initials: C
authors.lastname: Montoto
mesh_terms: D000119:Acetylmuramyl-Alanyl-Isoglutamine / Q000008:administration & dosage / Q000031:analogs & derivatives* / Q000191:economics; D000293:Adolescent; D000328:Adult; D000971:Antineoplastic Combined Chemotherapy Protocols / Q000008:administration & dosage* / Q000191:economics / Q000627:therapeutic use; D001859:Bone Neoplasms / Q000188:drug therapy* / Q000473:pathology; D002648:Child; D002675:Child, Preschool; D017326:Clinical Trials, Phase III as Topic; D003362:Cost-Benefit Analysis; D005260:Female; D006801:Humans; D007223:Infant; D008297:Male; D008390:Markov Chains; D009965:Orphan Drug Production / Q000191:economics; D012516:Osteosarcoma / Q000188:drug therapy* / Q000473:pathology; D010714:Phosphatidylethanolamines / Q000008:administration & dosage* / Q000191:economics; D019057:Quality-Adjusted Life Years; D013030:Spain; D055815:Young Adult
chemical_list: D010714:Phosphatidylethanolamines; C037144:mifamurtide; D000119:Acetylmuramyl-Alanyl-Isoglutamine
keywords: QALY; cost-effectiveness; mifamurtide; orphan drug; osteosarcoma; pharmacoeconomics