Tabelecleucel Medicine 12 June 2019 Extended indication Treatment of patients with Epstein-Barr Virus-associated Post Transplant Lymphoproliferative Disorder in the allogeneic hematopoietic cell transplant setting who have failed on rituximab. Therapeutic value No judgement Total cost Registration phase Clinical trials Product Active substance Tabelecleucel Domain Oncology and Hematology Reason of inclusion New medicine (specialité) Main indication Stem cell transplants Extended indication Treatment of patients with Epstein-Barr Virus-associated Post Transplant Lymphoproliferative Disorder in the allogeneic hematopoietic cell transplant setting who have failed on rituximab. Current proprietary name Already available biosimilars / generics Proprietary name Manufacturer Atara Portfolio holder Mechanism of action Allogeneic modified cell therapy Route of administration Intravenous Therapeutical formulation Intravenous drip Budgetting framework Intermural (MSZ) Centre of expertise Additional remarks Allogeneic Epstein-Barr virus-specific cytotoxic T lymphocytes. Registration Registration route Centralised (EMA) Type of trajectory Accelerated assessment Particularity New medicine with Priority Medicines (PRIME) ATMP Unknown Submission date 2020 Expected Registration 2021 Orphan drug Yes Registration phase Clinical trials Reimbursement Medicine sluice Additional remarks ATMP, PRIME Therapeutic value Current treatment options EMA: "At the time of designation, no satisfactory method were authorised in the European Union for the treatment of post-transplant lymphoproliferative disorder. Patients with the condition were treated with medicines authorised for non-Hodgkin’s lymphoma." Therapeutic value No judgement This assessment does not indicate any potential inclusion in the package. Substantiation Duration of treatment Frequency of administration 3 times every 5 weeks Dosage per administration References klinische studies: ALLELE en MATCH Additional remarks Tabelecleucel will be administered in cycles lasting 5 weeks (35 days). During each cycle, subjects will receive IV tabelecleucel at a dose of 2×10^6 cells/kg on Days 1, 8, and 15, followed by observation through Day 35. Participants may receive more than one cycle of therapy as required. Expected patient volume per year Patient volume Market share is generally not included unless otherwise stated. Maximum patient volume for sluice References Vektis Additional remarks Op basis van de add-on declaratiegegevens voor rituximab bij de indicatie: Behandeling van post-transplantatie lymfoproliferatieve ziekte (PTLD) na orgaan- of stamcel transplantatie bij volwassenen kan worden geconcludeerd dat er in 2017 ongeveer 30 patiënten dit middel hebben gekregen en in 2018 44 patiënten. De declaratiegegevens van 2018 zijn echter nog incompleet. Om die reden is de inschatting dat er zo'n 30 - 50 patiënten rituxmab krijgen bij PTLD. Gezien rituximab echter relatief goed werkt voor deze groep patiënten is de verwachting dat slechts een deel hiervan in aanmerking zal komen voor tabelecleucel. Expected cost per patient per year Cost References Additional remarks Potential total cost per year Total cost 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 Op dit moment zijn er geen fase 3 studies op andere indicatie-gebieden. References www.clinicaltrials.gov Additional remarks Other information Additional remarks