Sapacitabine Medicine 12 June 2018 Extended indication Acute myeloid leukemia Therapeutic value No judgement Total cost € 67,400,000.00 Registration phase Clinical trials Product Active substance Sapacitabine Domain Oncology and Hematology Reason of inclusion Main indication Leukemia Extended indication Acute myeloid leukemia Current proprietary name Already available biosimilars / generics Proprietary name Manufacturer Daiichi Sankyo Portfolio holder Mechanism of action Unknown Route of administration Oral Therapeutical formulation Capsule Budgetting framework Intermural (MSZ) Centre of expertise Additional remarks nucleoside analogue Registration Registration route Centralised (EMA) Type of trajectory Unknown Particularity New medicine ATMP Unknown Submission date 2019 Expected Registration 2020 Orphan drug Yes Registration phase Clinical trials Reimbursement Medicine sluice Additional remarks Fase III studie (NCT01303796) SEAMLESS: mixed results. Therapeutic value Current treatment options Decitabine Therapeutic value No judgement This assessment does not indicate any potential inclusion in the package. Substantiation Duration of treatment Frequency of administration Dosage per administration 400 mg References Kantarjian H. et al., Lancet Oncol. 2012 Nov;13(11):1096-104. doi: 10.1016/S1470-2045(12)70436-9. Additional remarks fase 2 studie laat zien dat 400 mg het meest effectief is en dat de toedieningsfrequentie als volgt is: 2 x per dag 3 dagen per week voor 2 weken in een cyclus van 21 dagen. Matige respons in: maximaal 30% 1 jaars overleving in groep oudere AML patienten, waarvan merendeel onbehandeld. Vooralsnog geen duidelijke plaats. Expected patient volume per year Patient volume < 674 Market share is generally not included unless otherwise stated. Maximum patient volume for sluice References NKR Additional remarks EMA: 'At the time of designation acute myeloid leukaemia affected 1 in 10,000 people in the European Union (EU).' Dat zou dus gaan om maximaal 1700 patienten in Nederland. Het NKR in 2015 geeft aan dat er 674 patienten zijn met AML. Expected cost per patient per year Cost € 100,000.00 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 Farmacotherapeutisch Kompas, medicijnkosten.nl Additional remarks kosten huidige behandeling Decitabine: € 1322,99 per 50 MG FL (medicijnkosten.nl), behandelplan Decitabine: i.v. 20 mg/m² lichaamsoppervlak/dag via infusie in 1 uur gedurende 5 achtereenvolgende dagen per behandelcyclus van 4 weken; minimaal 4 behandelcycli worden aanbevolen. De behandeling voortzetten tot aan ziekteprogressie of toxiciteit. (Bron: FK) Potential total cost per year Total cost € 67,400,000.00 Total cost for sluice Additional remarks Off label use Off label use Unknown Indications off label use References Additional remarks Indication extension Indication extensions No Indication extensions References Additional remarks Other information Additional remarks