Eculizumab Medicine 12 June 2018 Extended indication Neuromyelitis optica (NMO) relapsing; second-line or add-on therapy. Therapeutic value No judgement Total cost € 30,875,000.00 Registration phase Clinical trials Product Active substance Eculizumab Domain Chronic immune diseases Reason of inclusion Indication extension IND Main indication Eye disorders Extended indication Neuromyelitis optica (NMO) relapsing; second-line or add-on therapy. Current proprietary name Already available biosimilars / generics Proprietary name Soliris Manufacturer Alexion Portfolio holder Mechanism of action Unknown Route of administration Intravenous Therapeutical formulation Intravenous drip Budgetting framework Intermural (MSZ) Centre of expertise Additional remarks First in class, humanised monoclonal antibody C5 complement inhibitor. Registration Registration route Centralised (EMA) Type of trajectory Unknown Particularity Unknown ATMP Unknown Submission date 2019 Expected Registration March 2020 Orphan drug Yes Registration phase Clinical trials Reimbursement Medicine sluice Additional remarks Therapeutic value Current treatment options geen 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 References Additional remarks Expected patient volume per year Patient volume 65 Market share is generally not included unless otherwise stated. Maximum patient volume for sluice References Marrie and Gryba. Int J MS Care. 2013 Fall; 15(3): 113–118; Tackley et al. Mult Scler Relat Disord. 2016 May;7:21-5 Additional remarks Incidentie van NMO ligt rond de 0,4 per 100.000 inwoners, dit betekent voor Nederland ongeveer 70 patiënten. 93% van de patiënten ervaart een relapse wat neerkomt op 65. Expected cost per patient per year Cost € 450,000.00 - 500,000.00 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 Fabrikant; https://www.zorginstituutnederland.nl/publicaties/adviezen/2017/06/09/eculizumab-soliris-bij-pnh-herbeoordeling-pakketadvies Additional remarks Voor alle indicaties van eculizumab geldt een financieel arrangement dat afloopt per eind 2020. Voor een andere indicatie van eculizumab werd een bedrag van €360.000 euro per patiënt per jaar gerekend (excl. afspraken financieel arrangement). De fabrikant geeft aan dat gebaseerd op de lijstprijs en het behandelschema voor 12 maanden de prijs €450.000-500.000 zal bedragen. Potential total cost per year Total cost € 30,875,000.00 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 eculizumab biosimilar (ABP 959) · Paroxysmal nocturnal haemoglobinuria (PNH) and atypical hemolytic uremic syndrome (aHUS) References SPS Additional remarks Other information Additional remarks