LCAR-B38M CAR-T cell therapy Medicine 10 December 2019 Extended indication Adult patients with relapsed or refractory multiple myeloma, whose prior regimens included a proteasome inhibitor, an immunomodulatory agent and an anti-CD38 antibody and who had disease progression on the last regimen. Therapeutic value No judgement Total cost € 47,450,000.00 Registration phase Clinical trials Product Active substance LCAR-B38M CAR-T cell therapy Domain Oncology and Hematology Reason of inclusion New medicine (specialité) Main indication Stem cell transplants Extended indication Adult patients with relapsed or refractory multiple myeloma, whose prior regimens included a proteasome inhibitor, an immunomodulatory agent and an anti-CD38 antibody and who had disease progression on the last regimen. Current proprietary name Already available biosimilars / generics Proprietary name Manufacturer Janssen Portfolio holder Mechanism of action CAR-T therapy Route of administration Intravenous Therapeutical formulation Intravenous drip Budgetting framework Intermural (MSZ) Centre of expertise Additional remarks Autologe humane T cellen die ex-vivo genetisch gemodificeerd zijn met een lentivirale vector die codeert voor een chimeric antigen receptor (CAR) tegen het B-cell maturation antigen (BCMA). Registration Registration route Centralised (EMA) Type of trajectory Accelerated assessment Particularity New medicine with Priority Medicines (PRIME) ATMP Unknown Submission date January 2021 Expected Registration August 2021 Orphan drug Unknown Registration phase Clinical trials Reimbursement Medicine sluice Additional remarks Dit geneesmiddel is geclassificeerd als een ATMP. Volgens fabrikant registratie in Q3 2021. 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 NCT03548207 Additional remarks Expected patient volume per year Patient volume 125 - 166 Market share is generally not included unless otherwise stated. Maximum patient volume for sluice References Pakketadvies daratumumab; Yong et al. Br J Haematol. 2016 Oct;175(2):252-264; Willenbacher et al. PLoS One. 2016 Mar 3;11(3):e0147381 Additional remarks Uit het pakketadvies voor daratumumab blijkt dat 830 patiënten in de eerste lijn behandeld worden. Het geschatte aantal patiënten wat in de derde lijn behandeld wordt voor multiple myeloom is 418. Van het totaal van 830 patiënten zal op basis van de literatuur ongeveer 15-20% in de 4e lijn behandeld, dit zou dus neerkomen op 125-166 patiënten. Expected cost per patient per year Cost € 325,000.00 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 Additional remarks De officiële prijs is nog niet bekend. Mogelijk zijn de kosten in lijn met andere CAR-T therapieën die rond de €325.000 kosten. Potential total cost per year Total cost € 47,450,000.00 Total cost for sluice Additional remarks Off label use Off label use Unknown Indications off label use References Additional remarks Indication extension Indication extensions No Indication extensions References adisinsight Additional remarks Other information Additional remarks