Tocilizumab Medicine 13 December 2018 Extended indication RoActemra is indicated for the treatment of active systemic juvenile idiopathic arthritis (sJIA) in patients 1 year of age and older, who have responded inadequately to previous therapy with NSAIDs and systemic corticosteroids. RoActemra can be given as monotherapy (in case of intolerance to MTX or where treatment with MTX is inappropriate) or in combination with MTX. Therapeutic value No judgement Total cost € 165,750.00 Registration phase Registered and reimbursed Product Active substance Tocilizumab Domain Chronic immune diseases Reason of inclusion Indication extension IND Main indication Rheumatism Extended indication RoActemra is indicated for the treatment of active systemic juvenile idiopathic arthritis (sJIA) in patients 1 year of age and older, who have responded inadequately to previous therapy with NSAIDs and systemic corticosteroids. RoActemra can be given as monotherapy (in case of intolerance to MTX or where treatment with MTX is inappropriate) or in combination with MTX. 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 Additional remarks Monoklonaal antilichaam dat de werking van interleukine 6 (IL-6) blokkeert. Registration Registration route Centralised (EMA) Type of trajectory Normal trajectory Particularity Unknown ATMP Unknown Submission date April 2018 Expected Registration November 2018 Orphan drug No Registration phase Registered and reimbursed Reimbursement Medicine sluice Additional remarks Positieve CHMP-opinie september 2018. Geregistreerd in november 2018. Therapeutic value Current treatment options NSAIDs and systemic corticosteroids Therapeutic value No judgement This assessment does not indicate any potential inclusion in the package. Substantiation Duration of treatment Frequency of administration 1 times every 2 weeks Dosage per administration 12 mg/kg References Farmacotherapeutisch Kompas; EPAR Additional remarks Expected patient volume per year Patient volume < 17 Market share is generally not included unless otherwise stated. Maximum patient volume for sluice References ReumaNederland; CBS; ErasmusMC kindergeneeskunde JIA Additional remarks Jeugdreuma komt voor bij ongeveer 1:1000 kinderen. In Nederland waren in 2017 ongeveer 170.000 kinderen tussen 1-2 jaar oud. Dit zijn dus ongeveer 170 kinderen. Systemische Juveniele Idiopathische Artritis beslaat ongeveer 10% van de gevallen van jeugdreuma en dus 17 patiënten. Hiervan zal tocilizumab alleen in de tweede lijn worden gebruikt. Expected cost per patient per year Cost € 8,500.00 - 11,000.00 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 Een flacon van 4 ml tocilizumab kost €163,90 en een flacon van 10 ml tocilizumab kost €409,76. Voor een kind van 10-15 kg is er per injectie 120-180 mg tocilizumab nodig. Dit zijn 2 flacons van 4 ml voor een kind van 10 kg en 1 flacon van 10 ml voor een kind van 15 kg. Voor de behandeling van een jaar betekend dit 52 flacons van 4 ml voor een kind van 10 kg en 26 flacons van 10 ml voor een kind van 15 kg. In totaal kost een behandeling in deze range dus €8.522,65-€10.654,14. Potential total cost per year Total cost € 165,750.00 Total cost for sluice Additional remarks Off label use Off label use Yes Indications off label use References Roche pipeline Additional remarks Indication extension Indication extensions Unknown Indication extensions References Additional remarks Other information Additional remarks