Treosulfan Medicine 12 June 2018 Extended indication Treosulfan in combination with fludarabine is indicated as part of conditioning treatment prior to allogeneic haematopoietic stem cell transplantation (alloHSCT) in adult patients with malignant and non-malignant diseases, and in paediatric patients older than one month with malignant diseases. Therapeutic value No judgement Total cost € 671,000.00 Registration phase Registered Product Active substance Treosulfan Domain Oncology and Hematology Reason of inclusion New medicine (specialité) Main indication Stem cell transplants Extended indication Treosulfan in combination with fludarabine is indicated as part of conditioning treatment prior to allogeneic haematopoietic stem cell transplantation (alloHSCT) in adult patients with malignant and non-malignant diseases, and in paediatric patients older than one month with malignant diseases. Current proprietary name Already available biosimilars / generics Proprietary name Trecondi Manufacturer Medac Portfolio holder Mechanism of action Alkylating agent 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 Unknown ATMP Unknown Submission date February 2018 Expected Registration June 2019 Orphan drug Yes Registration phase Registered Reimbursement Medicine sluice Additional remarks Positieve CHMP-opinie december 2018. Geregistreerd in juni 2019. 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. Inmiddels is de prijs van Treosulfan bekend: injectieflacon peoder oplossing voor infusie van 1.000MG FL: €95,68 Uitgaande van gemiddeld 3 behandeldagen en een persoon van 1,8m2 kom je uit op €5.166,73 per patiënt per jaar. 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