Tisagenlecleucel Medicine 09 January 2018 Extended indication Paediatric Acute Lymphatic Leukeamia (ALL): relapse and/or refractory patients, not eligible for SCT (3L). Therapeutic value Possible added value Total cost € 4,750,000.00 Registration phase Unknown Product Active substance Tisagenlecleucel Domain Oncology and Hematology Reason of inclusion Main indication Leukemia Extended indication Paediatric Acute Lymphatic Leukeamia (ALL): relapse and/or refractory patients, not eligible for SCT (3L). Current proprietary name Already available biosimilars / generics Proprietary name Kymriah Manufacturer Novartis Portfolio holder Mechanism of action Unknown Route of administration Intravenous Therapeutical formulation Intravenous drip Budgetting framework Intermural (MSZ) Centre of expertise Additional remarks Registration Registration route Centralised (EMA) Type of trajectory Accelerated assessment Particularity Unknown ATMP Unknown Submission date December 2017 Expected Registration August 2018 Orphan drug Yes Registration phase Unknown Reimbursement Medicine sluice Additional remarks PRIME granted, EMA 23-06-2016. Therapeutic value Current treatment options Stamceltransplantatie, chemotherapie Therapeutic value Possible added value This assessment does not indicate any potential inclusion in the package. Substantiation SKION protocol ALL-11: Treatment study protocol of the Dutch Childhood Oncology Group for children and adolescents (1-19 year) with newly diagnosed acute lymphoblastic leukemia Duration of treatment Average 1 day / days Frequency of administration 1 times a day Dosage per administration 1 dosis References Additional remarks Eénmalige infusie, Advanced Therapy Medicinal Products (ATMP) gentherapie. Expected patient volume per year Patient volume < 10 Market share is generally not included unless otherwise stated. Maximum patient volume for sluice References Fabrikant Additional remarks Fabrikant: Momenteel is er nog weinig ervaring met behandeling van patiënten met ATMP's. Hierdoor is het momenteel niet duidelijk hoe de ATMP's in de dagelijkse praktijk zullen worden ingezet. Realistisch aantal patiënten: onbekend, afhankelijk van studieresultaten en label. Waarschijnlijk zeer klein aantal patiënten. Veld: uiteindelijk veel meer patienten. Expected cost per patient per year Cost € < 475,000.00 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 Additional remarks Meerkosten. Kymriah is in the VS voor de pALL indictie goedgekeurd. Prijs in de VS vastgesteld op $475.000. Echter, dit zijn alleen de kosten voor het preparaat; hier komen nog kosten bij voor leukaferese, opname voor toediening en opvangen toxiciteit, inclusief toediening andere dure geneesmiddelen als tocilizumab, eventuele IC opname en eventuele langdurige gammaglobuline-suppletie. Potential total cost per year Total cost € 4,750,000.00 Total cost for sluice Additional remarks Off label use Off label use Unknown Indications off label use References Additional remarks Indication extension Indication extensions Unknown Indication extensions References Additional remarks Other information Additional remarks