Emicizumab Medicine 09 January 2018 Extended indication Prophylaxis to prevent bleeding or reduce the frequency of bleeding episodes in adults and children with hemophilia A (congenital factor VIII deficiency) with factor VIII inhibitors. Therapeutic value Possible added value Total cost € 19,665,000.00 Registration phase Unknown Product Active substance Emicizumab Domain Oncology and Hematology Reason of inclusion Main indication Haemophilia Extended indication Prophylaxis to prevent bleeding or reduce the frequency of bleeding episodes in adults and children with hemophilia A (congenital factor VIII deficiency) with factor VIII inhibitors. Current proprietary name Already available biosimilars / generics Proprietary name Hemlibra Manufacturer Roche Portfolio holder Mechanism of action Unknown Route of administration Subcutaneous Therapeutical formulation Injection Budgetting framework Intermural (MSZ) Centre of expertise Hemofilie Behandel Centra Additional remarks Registration Registration route Centralised (EMA) Type of trajectory Normal trajectory Particularity Unknown ATMP Unknown Submission date August 2017 Expected Registration March 2018 Orphan drug Yes Registration phase Unknown Reimbursement Medicine sluice Additional remarks Therapeutic value Current treatment options FEIBA en recombinant factor VIIa Therapeutic value Possible added value This assessment does not indicate any potential inclusion in the package. Substantiation Duration of treatment Frequency of administration 1 times a week Dosage per administration 3 mg/kg References Additional remarks Fabrikant: Chronische behandeling. Dosering is conform klinische studie, kan volgens officiële SmPC nog veranderen. Week 1-4: 3 mg/kg; week ≥ 5: 1,5 mg/kg Expected patient volume per year Patient volume 13 - 100 Market share is generally not included unless otherwise stated. Maximum patient volume for sluice References Additional remarks Emicuzumab onderhoud is aangewezen voor deel van patienten bij wie remmer niet verdwijnt na ITI. Percentage patiënten is waarschijnlijk lager dan 20% (formele registratie ontbreekt nog), dan zou het totaal aantal maximaal uitkomen op 100, en het aantal dat daadwerkelijk behandeld zal worden op 25-50. Expected cost per patient per year Cost € 170,000.00 - 520,000.00 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 Additional remarks Berekend obv de prijs voor octocog alfa en behandelschema beschreven in het farmacotherapeutisch kompas voor een patient van 70 kg langetermijnprofilaxe: €170.000-520.000. schatting gedaan op basis van de huidige behandelopties waarmee het moet concurreren. Potential total cost per year Total cost € 19,665,000.00 Total cost for sluice Additional remarks Off label use Off label use No Indications off label use References Additional remarks Indication extension Indication extensions No Indication extensions References Additional remarks Other information Additional remarks