Rurioctocog alfa pegol Medicine 09 January 2018 Extended indication Haemophilia A; severe in adults and children from 0 to 75 years, Second or subsequent line, for treatment and prevention of bleeding, routine prophylaxis and perioperative bleeding. Therapeutic value No judgement Total cost € 164,937,500.00 Registration phase Unknown Product Active substance Rurioctocog alfa pegol Domain Oncology and Hematology Reason of inclusion Main indication Haemophilia Extended indication Haemophilia A; severe in adults and children from 0 to 75 years, Second or subsequent line, for treatment and prevention of bleeding, routine prophylaxis and perioperative bleeding. Current proprietary name Already available biosimilars / generics Proprietary name Adynovi Manufacturer Shire Portfolio holder Mechanism of action Unknown Route of administration Intravenous Therapeutical formulation Injection Budgetting framework Intermural (MSZ) Centre of expertise Additional remarks PEGylated versie van Advate. Registration Registration route Centralised (EMA) Type of trajectory Normal trajectory Particularity Unknown ATMP Unknown Submission date April 2016 Expected Registration January 2018 Orphan drug No Registration phase Unknown Reimbursement Medicine sluice Additional remarks Positieve CHMP opinie november 2017. Therapeutic value Current treatment options Verschillende recombinante factor VIII eiwitten. Therapeutic value No judgement This assessment does not indicate any potential inclusion in the package. Substantiation Duration of treatment Frequency of administration 3 times a week Dosage per administration 20-40 IE/kg References Farmacotherapeutisch kompas Additional remarks Toediningsfrequentie: 1 maal per 2-3 dagen; Obv langetermijnprofylaxe beschreven in FK. Expected patient volume per year Patient volume < 725 Market share is generally not included unless otherwise stated. Maximum patient volume for sluice References Additional remarks Max. patiënten: Ongeveer 1.450 patiënten hebben hemofilie A. De helft hiervan heeft een ziektestaat die moderate/severe is en wordt behandeld met factor VIII. Realistische patientenaantallen lastig in te schatten. Expected cost per patient per year Cost € 110,000.00 - 345,000.00 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; NVHP; Fabrikant Additional remarks Berekend obv de prijs voor octocog alfa via medicijnkosten.nl en behandelschema beschreven in het farmacotherapeutisch kompas voor een patient van 70 kg langetermijnprofilaxe: €170.000-520.000. De nederlands vereniging voor hemofiliepatiënten geeft aan dat alle hemofiliebehandelingen in 2014 in totaal €135 miljoen kostten (voor 1600 hemofiliepatienten). Fabrikant (nov 2017): 110.000-345.000 p.p.p.j. Potential total cost per year Total cost € 164,937,500.00 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