Eculizumab Medicine 09 January 2018 Extended indication Refractory generalized myasthenia gravis (gMG) in patients who are anti-acetylcholine receptor (AChR) antibody-positive. Therapeutic value No judgement Total cost € 612,000,000.00 Registration phase Unknown Product Active substance Eculizumab Domain Neurological disorders Reason of inclusion Main indication Muscular diseases Extended indication Refractory generalized myasthenia gravis (gMG) in patients who are anti-acetylcholine receptor (AChR) antibody-positive. 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 Registration Registration route Centralised (EMA) Type of trajectory Normal trajectory Particularity Unknown ATMP Unknown Submission date Expected Registration August 2017 Orphan drug Yes Registration phase Unknown Reimbursement Medicine sluice Additional remarks Betreft een indicatie-uitbreiding. Geregistreerd in augustus 2017. Soliris is al geregistreerd voor indicatie PNH en aHUS. Therapeutic value Current treatment options 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 < 1,700 Market share is generally not included unless otherwise stated. Maximum patient volume for sluice References https://www.spierziekten.nl/fileadmin/user_upload/VSN/documenten/Huisartsenbrochures/M010-MG-habrochmetbrieven.pdf Additional remarks 1 patiënt per 10.000 inwoners Expected cost per patient per year Cost € 360,000.00 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 Zorginstituut Nederland, Prescribing Outlook 2017. Additional remarks Zorginstituut: De kosten per patiënt per jaar bedragen voor eculizumab circa € 360.000. Rekening houdend met deze geneesmiddelkosten wordt het totale kostenbeslag, ondanks de kleine groep patiënten, geschat op € 25 miljoen in 2017. Prescribing Outlook 2017: 'Extremely expensive. At list price, cost is £352,800 per year; but alternative options are also expensive.' Potential total cost per year Total cost € 612,000,000.00 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