Treosulfan Medicine 12 June 2018 Extended indication Conditioning treatment prior to haematopoietic progenitor cell transplantation Therapeutic value No judgement Total cost € 671,000.00 Registration phase Registration application pending Product Active substance Treosulfan Domain Oncology and Hematology Reason of inclusion Main indication Other hematology Extended indication Conditioning treatment prior to haematopoietic progenitor cell transplantation Current proprietary name Already available biosimilars / generics Proprietary name Manufacturer Medac Portfolio holder Mechanism of action Unknown Route of administration Intravenous Therapeutical formulation Intravenous drip Budgetting framework Intermural (MSZ) Centre of expertise Additional remarks Alkylerend middel Registration Registration route Centralised (EMA) Type of trajectory Normal trajectory Particularity New medicine ATMP Unknown Submission date February 2018 Expected Registration March 2019 Orphan drug Yes Registration phase Registration application pending Reimbursement Medicine sluice Additional remarks Therapeutic value Current treatment options Busulfan Therapeutic value No judgement This assessment does not indicate any potential inclusion in the package. Substantiation Duration of treatment Average 3 day / days Frequency of administration 1 times a day Dosage per administration 10 g/m2 References NCT00822393 Additional remarks Toedieningen op dagen -4, -3 en -2. Expected patient volume per year Patient volume < 122 Market share is generally not included unless otherwise stated. Maximum patient volume for sluice References GIPdatabank Additional remarks 122 gebruikers van busulfan in 2015. Expected cost per patient per year Cost € 5,000.00 - 6,000.00 This amount gives an indication of the total cost. It is the result of the average expected patient volume times the average cost per patient. both per year. References Medicijnkosten.nl; Vademecum Hematologie Additional remarks Busulfan volgens behandelschema vademecum hematologie voor een patient van 70 kg: 19 flacons, in totaal €5.883,70. Potential total cost per year Total cost € 671,000.00 Total cost for sluice Additional remarks Off label use Off label use Unknown Indications off label use References Additional remarks Indication extension Indication extensions Yes Indication extensions Ovarian cancer, ewing sarcoma, ALL. References Clinicaltrials.gov Additional remarks Other information Additional remarks