Tocilizumab Medicine 12 June 2018 Extended indication Systemische Sclerose Therapeutic value No judgement yet Total cost € 525,000.00 Registration phase Registration application pending Product Active substance Tocilizumab Domain Chronic immune diseases Reason of inclusion Indication extension IND Main indication Other chronic immune diseases Extended indication Systemische Sclerose Current proprietary name Already available biosimilars / generics Proprietary name RoActemra Manufacturer Roche Portfolio holder Mechanism of action Unknown Route of administration Subcutaneous Therapeutical formulation Injection Budgetting framework Intermural (MSZ) Centre of expertise Expertise centra voor systemische sclerose. Additional remarks Monoklonaal antilichaam dat de werking van interleukine 6 blokkeert. Registration Registration route Centralised (EMA) Type of trajectory Normal trajectory Particularity Unknown ATMP Unknown Submission date July 2018 Expected Registration August 2019 Orphan drug No Registration phase Registration application pending Reimbursement Medicine sluice Additional remarks Therapeutic value Current treatment options Er is momenteel geen behandeling Therapeutic value No judgement yet This assessment does not indicate any potential inclusion in the package. Substantiation Fase II studie heeft de eindpunten niet heeft gehaald maar is wel gepubliceerd (Khanna et al. Lancet. 2016 Jun 25;387(10038):2630-2640). Momenteel is een fase III studie gaande. Waarschijnlijk vanwege de unmet need. Een meer recente studie in ARD (Khanna et al. Ann Rheum Dis. 2018 Feb;77(2):212-220) - een open-label extension studie van het Lancet artikel - is positief maar er is kritiek op de statistiek. Fase III studie is gestart in augustus 2017. Duration of treatment Frequency of administration 1 times a week Dosage per administration 162 mg References NCT01532869 Additional remarks Expected patient volume per year Patient volume < 30 Market share is generally not included unless otherwise stated. Maximum patient volume for sluice References Additional remarks Gebruik wordt mogelijk beperkt tot expertisecentra. Zal naar verwachting minder dan 30 patiënten per jaar zijn. In totaal zijn er zijn momenteel 2.500 patiënten in Nederland bekend. Expected cost per patient per year Cost € 15,000.00 - 20,000.00 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 € 155 per 80 mg flacon Potential total cost per year Total cost € 525,000.00 Total cost for sluice Additional remarks Off label use Off label use Yes Indications off label use References Additional remarks Indication extension Indication extensions Unknown Indication extensions References Additional remarks Other information Additional remarks