title: [Cost per responder associated with romiplostim and rituximab treatment for adult primary immune thrombocytopenia in France].
abstract: OBJECTIVE
  This analysis compared the response rates and cost per responder associated with romiplostim and rituximab in adult immune thrombocytopenia from the French National Health System payer perspective.
  
  
  METHODS
  A decision analytic model was developed to estimate the cost per patient and per responder of treating adult immune thrombocytopenia patients with romiplostim versus rituximab over 6 months. A systematic literature review identified phase 3 randomized controlled trials. Published response rates were extracted (response definition: ≥50×10(9) platelets/liter). Resource utilization was based on French and international treatment guidelines, and clinical expert opinion. Unit costs were derived from literature and French reimbursement lists, and included the costs of routine physician visits, treatment administration, and emergency care. Non-responders incurred bleeding-related event costs.
  
  
  RESULTS
  The literature review identified a phase 3 randomized controlled trial for romiplostim with a response rate of 83%. Due to a lack of phase 3 randomized controlled trials for rituximab, a systematic review of studies was selected as the best source, reporting a response rate of 62.5%. Romiplostim and rituximab were associated with similar treatment costs, with an estimated cost per patient for romiplostim of €17,456 and €17,068 for rituximab. Rituximab resulted in a 30% higher cost per responder (€27,308 for rituximab versus €21,031 for romiplostim). Romiplostim use reduced drug administration, intravenous immunoglobulin, and bleeding-related hospitalization costs compared to rituximab.
  
  
  CONCLUSIONS
  Due to its high efficacy leading to lower bleeding-related costs, romiplostim represents an efficient use of resources for adult immune thrombocytopenia patients in the French healthcare system.
authors.affiliation: Service de médecine interne, centre de compétence PACA ouest pour la prise en charge des cytopénies autoimmunes, hôpital de la Conception, Aix-Marseille université, 13005 Marseille, France.
authors.forename: L
authors.initials: L
authors.lastname: Chiche
authors.affiliation: LA-SER Analytica, 24, West 40th Street, Floor 8, 10018 New York, États-Unis. Electronic address: allison.perrin@la-ser.com.
authors.forename: A
authors.initials: A
authors.lastname: Perrin
authors.affiliation: LA-SER Analytica, 24, West 40th Street, Floor 8, 10018 New York, États-Unis.
authors.forename: L
authors.initials: L
authors.lastname: Stern
authors.affiliation: Amgen (Europe) GmbH, Dammstrasse 23, Opus 105, 6301 Zug, Suisse.
authors.forename: L
authors.initials: L
authors.lastname: Kutikova
authors.affiliation: Amgen SAS, Building B, 62/64 boulevard Victor-Hugo, 92200 Neuilly, France.
authors.forename: S
authors.initials: S
authors.lastname: Cohen-Nizard
authors.affiliation: Service de biothérapie, hôpital Necker, 149, rue de Sèvres, 75015 Paris, France.
authors.forename: F
authors.initials: F
authors.lastname: Lefrère
mesh_terms: D000328:Adult; D058846:Antibodies, Monoclonal, Murine-Derived / Q000191:economics* / Q000627:therapeutic use; D003661:Decision Support Techniques; D016527:Drug Costs; D005602:France; D006801:Humans; D007155:Immunologic Factors / Q000191:economics* / Q000627:therapeutic use*; D018803:Models, Economic; D016553:Purpura, Thrombocytopenic, Idiopathic / Q000188:drug therapy / Q000191:economics*; D011961:Receptors, Fc / Q000627:therapeutic use; D053628:Receptors, Thrombopoietin / Q000819:agonists*; D011993:Recombinant Fusion Proteins / Q000191:economics* / Q000627:therapeutic use; D000069283:Rituximab; D013926:Thrombopoietin / Q000191:economics* / Q000627:therapeutic use; D016896:Treatment Outcome
chemical_list: D058846:Antibodies, Monoclonal, Murine-Derived; D007155:Immunologic Factors; D011961:Receptors, Fc; D053628:Receptors, Thrombopoietin; D011993:Recombinant Fusion Proteins; D000069283:Rituximab; D013926:Thrombopoietin; C488777:romiplostim
keywords: Adult primary immune thrombocytopenia (PTI); Economic model; Modèle économique; Rituximab; Romiplostim; Thrombocytopénie immune primaire chez l’adulte