Tocilizumab Medicine 12 June 2018 Extended indication Extension of indication to include treatment of new indication for slowing the rate of decline in pulmonary function in adult patients with systemic sclerosis-associated interstitial lung disease (SSc-ILD) for RoActemra. Therapeutic value No estimate possible yet Total cost € 5,250,000.00 Registration phase No registration expected Product Active substance Tocilizumab Domain Chronic immune diseases Reason of inclusion Indication extension IND Main indication Other chronic immune diseases Extended indication Extension of indication to include treatment of new indication for slowing the rate of decline in pulmonary function in adult patients with systemic sclerosis-associated interstitial lung disease (SSc-ILD) for RoActemra. Current proprietary name Already available biosimilars / generics Proprietary name RoActemra Manufacturer Roche Portfolio holder Mechanism of action Interleukin inhibitor 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 No Submission date July 2018 Expected Registration Orphan drug No Registration phase No registration expected Reimbursement Medicine sluice Additional remarks Registratieaanvraag teruggetrokken september 2023 Therapeutic value Current treatment options Er is momenteel geen effectieve behandeling Therapeutic value No estimate possible 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). Er was 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 was kritiek op de statistiek. Fase III studie is gestart in augustus 2017. In het derde kwartaal van 2018 heeft Roche de ontwikkeling stopgezet. De primaire eindpunten werden niet gehaald en er was een verhoogde kans op infecties. Duration of treatment Frequency of administration 1 times a week Dosage per administration 162 mg References NCT02453256 Additional remarks Expected patient volume per year Patient volume 200 - 400 Market share is generally not included unless otherwise stated. Maximum patient volume for sluice References Additional remarks Gebruik wordt mogelijk beperkt tot expertisecentra. In totaal zijn er zijn momenteel 2.500 patiënten in Nederland bekend. De werkgroep verwacht dat er naar schatting 200-400 patiënten in aanmerking zouden komen voor deze behandeling. 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 € 5,250,000.00 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