title: The budget impact and cost-effectiveness of defibrotide for treatment of veno-occlusive disease with multi-organ dysfunction in patients post-hematopoietic stem cell transplant.
abstract: BACKGROUND
  A Phase-3 study of defibrotide compared with historical controls demonstrated a 23% improvement in 100-day survival post-hematopoietic stem cell transplantation (HSCT) among patients with veno-occlusive disease with multi-organ dysfunction (VOD with MOD).
  
  
  OBJECTIVE
  To estimate the budget impact and cost-effectiveness of introducing defibrotide to a transplant center.
  
  
  METHODS
  The authors developed a budget impact model from the perspective of a bone-marrow transplant center. It was estimated that 2.3% of adults and 4.2% of children would develop VOD with MOD following HSCT based on a retrospective hospital database analysis and the effect that treating patients with defibrotide would have on costs for adult and pediatric centers was estimated. A cost-utility analysis (CUA) was also developed to capture the long-term cost-effectiveness of defibrotide. Projected life expectancies in the two groups were estimated based on trial data, transplant registry data, studies of long-term survival among HSCT patients, and US population life-tables.
  
  
  RESULTS
  There was an estimated 3% increase ($330,706) per year in total adult transplantation center costs associated with adopting defibrotide, and a <1% increase ($106,385) for pediatric transplant centers, assuming 100 transplants per year. In the CUA, the lifetime increase in cost per patient was $106,928, life expectancy increased by 3.74 years, and quality-adjusted life-years (QALYs) increased by 2.24. The incremental cost-effectiveness ratio (ICER) was $47,736 per QALY gained; 88% probability defibrotide was cost-effective at a $100,000/QALY threshold.
  
  
  CONCLUSIONS
  The budget impact of defibrotide for a transplant center is relatively modest compared to the overall cost of transplantation. Defibrotide provides an important survival advantage for VOD with MOD patients, and the life years gained lead to defibrotide being highly cost-effective.
authors.affiliation: a University of Washington , Seattle , WA , USA.
authors.forename: David L
authors.initials: DL
authors.lastname: Veenstra
authors.affiliation: a University of Washington , Seattle , WA , USA.
authors.forename: Gregory F
authors.initials: GF
authors.lastname: Guzauskas
authors.affiliation: b Jazz Pharmaceuticals, Inc. , Palo Alto , CA.
authors.forename: Kathleen F
authors.initials: KF
authors.lastname: Villa
authors.affiliation: a University of Washington , Seattle , WA , USA.
authors.forename: Denise M
authors.initials: DM
authors.lastname: Boudreau
mesh_terms: D002017:Budgets / Q000706:statistics & numerical data*; D003362:Cost-Benefit Analysis; D005343:Fibrinolytic Agents / Q000191:economics* / Q000627:therapeutic use; D018380:Hematopoietic Stem Cell Transplantation / Q000009:adverse effects; D006504:Hepatic Veno-Occlusive Disease / Q000191:economics / Q000209:etiology / Q000628:therapy*; D006801:Humans; D017059:Models, Econometric; D009102:Multiple Organ Failure / Q000191:economics / Q000209:etiology / Q000628:therapy*; D011089:Polydeoxyribonucleotides / Q000191:economics* / Q000627:therapeutic use; D019057:Quality-Adjusted Life Years; D012189:Retrospective Studies; D016019:Survival Analysis; D014481:United States
chemical_list: D005343:Fibrinolytic Agents; D011089:Polydeoxyribonucleotides; C036901:defibrotide
keywords: Budget impact; Cost; Cost-effectiveness; Decision analysis; Defibrotide; Sinusoidal obstruction syndrome; Veno-occlusive disease