title: Defibrotide in Severe Sinusoidal Obstruction Syndrome: Medicine and Economic Issues.
abstract: In Europe, Defitelio (defibrotide) has a Market Authorization in curative treatment of severe sinusoidal obstruction syndrome (SOS) but not in prophylaxis (2013). In France, defibrotide has had a compassionate-use program since 2009. Today, the high cost of defibrotide remains a major hurdle for hospital budgets. Medicine and economic issues were evaluated for the 39 hospitals of the French Public Assistance-Hospitals of Paris (AP-HP). We analyzed literature reviews, consumption, and expenditures through AP-HP data in 2014 and patient profiles with defibrotide in the corresponding diagnostic-related groups (DRGs) and consulted a board of hematologists. Finally, 18 publications were selected. Between 2011 and 2014 consumption increased to €5.2M. In 2014, 80 patients receiving defibrotide were mainly ascribed to the DRG "hematopoietic stem cell transplantation" levels 3 or 4. The tariffs attributed to drugs (€3544 to 4084) cover a small part of treatment costs (€97,524 for an adult). French experts thus recommended a harmonization of indications in prophylaxis (off-label use), improvement of pretransplant care, and optimization of the number of vials used. The economic impact led experts to change their practices. They recommended the restriction of defibrotide use to SOS curative treatment and to high-risk situations in prophylaxis.
authors.affiliation: General Agency of Equipment and Health Products (AGEPS), Assistance Publique-Hôpitaux de Paris (AP-HP), Paris, France. Electronic address: juliestee@gmail.com.
authors.forename: Julie
authors.initials: J
authors.lastname: Steelandt
authors.affiliation: General Agency of Equipment and Health Products (AGEPS), Assistance Publique-Hôpitaux de Paris (AP-HP), Paris, France; Health Law and Health Economics Department, Faculty of Pharmacy, Paris Descartes University, Sorbonne Paris Cité, Paris, France; Health Law Institute, Inserm, UMR S 1145, Paris Descartes University, Sorbonne Paris Cité, Paris, France.
authors.forename: François
authors.initials: F
authors.lastname: Bocquet
authors.affiliation: General Agency of Equipment and Health Products (AGEPS), Assistance Publique-Hôpitaux de Paris (AP-HP), Paris, France.
authors.forename: Anne-Laure
authors.initials: AL
authors.lastname: Cordonnier
authors.affiliation: General Agency of Equipment and Health Products (AGEPS), Assistance Publique-Hôpitaux de Paris (AP-HP), Paris, France.
authors.forename: Charlotte
authors.initials: C
authors.lastname: De Courtivron
authors.affiliation: General Agency of Equipment and Health Products (AGEPS), Assistance Publique-Hôpitaux de Paris (AP-HP), Paris, France.
authors.forename: Isabelle
authors.initials: I
authors.lastname: Fusier
authors.affiliation: General Agency of Equipment and Health Products (AGEPS), Assistance Publique-Hôpitaux de Paris (AP-HP), Paris, France; Health Law and Health Economics Department, Faculty of Pharmacy, Paris Descartes University, Sorbonne Paris Cité, Paris, France; Health Law Institute, Inserm, UMR S 1145, Paris Descartes University, Sorbonne Paris Cité, Paris, France.
authors.forename: Pascal
authors.initials: P
authors.lastname: Paubel
mesh_terms: D000328:Adult; D002017:Budgets; D002648:Child; D002986:Clinical Trials as Topic; D015331:Cohort Studies; D016527:Drug Costs; D016903:Drug Monitoring; D005343:Fibrinolytic Agents / Q000191:economics / Q000627:therapeutic use*; D005602:France; D018380:Hematopoietic Stem Cell Transplantation / Q000009:adverse effects; D006504:Hepatic Veno-Occlusive Disease / Q000188:drug therapy* / Q000191:economics / Q000453:epidemiology; D006779:Hospitals, Public; D006801:Humans; D015337:Multicenter Studies as Topic; D056687:Off-Label Use; D011089:Polydeoxyribonucleotides / Q000191:economics / Q000627:therapeutic use*; D017410:Practice Guidelines as Topic; D015995:Prevalence; D012189:Retrospective Studies
chemical_list: D005343:Fibrinolytic Agents; D011089:Polydeoxyribonucleotides; C036901:defibrotide
keywords: Budget impact analysis; Defibrotide; Off-label use; Scientific evaluation; Sinusoidal obstruction syndrome