Plerixafor Medicine 13 December 2018 Extended indication Extension of Indication to include paediatric patients aged 1 to 18 years. Mozobil is indicated in combination with G-CSF to enhance mobilisation of haematopoietic stem cells to the peripheral blood for collection and subsequent autologous transplantation in children with lymphoma or solid malignant tumours, either: • pre-emptively, when circulating stem cell count on the predicted day of collection after adequate mobilization with G-CSF (with or without chemotherapy) is expected to be insufficient with regards to desired hematopoietic stem cells yield, or • who previously failed to collect sufficient haematopoietic stem cells. Therapeutic value No judgement Total cost € 88,000.00 Registration phase Registered and reimbursed Product Active substance Plerixafor Domain Oncology and Hematology Reason of inclusion Indication extension IND Main indication Stem cell transplants Extended indication Extension of Indication to include paediatric patients aged 1 to 18 years. Mozobil is indicated in combination with G-CSF to enhance mobilisation of haematopoietic stem cells to the peripheral blood for collection and subsequent autologous transplantation in children with lymphoma or solid malignant tumours, either: • pre-emptively, when circulating stem cell count on the predicted day of collection after adequate mobilization with G-CSF (with or without chemotherapy) is expected to be insufficient with regards to desired hematopoietic stem cells yield, or • who previously failed to collect sufficient haematopoietic stem cells. Current proprietary name Already available biosimilars / generics Proprietary name Mozobil Manufacturer Genzyme Portfolio holder Mechanism of action Receptor antagonist Route of administration Subcutaneous Therapeutical formulation Injection Budgetting framework Intermural (MSZ) Centre of expertise Additional remarks Alpha chemokine receptor CXCR4 antagonist. Registration Registration route Centralised (EMA) Type of trajectory Normal trajectory Particularity Unknown ATMP Unknown Submission date March 2018 Expected Registration May 2019 Orphan drug Yes Registration phase Registered and reimbursed Reimbursement Medicine sluice Additional remarks Positieve CHMP-opinie maart 2019. Therapeutic value Current treatment options Oogsten met GCSF alleen Therapeutic value No judgement This assessment does not indicate any potential inclusion in the package. Substantiation Duration of treatment Frequency of administration 1 times a day Dosage per administration 0,24 mg/kg References EPAR Additional remarks Plerixafor dient 6 tot 11 uur vóór aanvang van elke aferese te worden toegediend door middel van een subcutane injectie, na een voorbehandeling van 4 dagen met G-CSF. Bij klinische onderzoeken werd plerixafor meestal gedurende 2 tot 4 (en tot maximaal 7) opeenvolgende dagen gebruikt. Expected patient volume per year Patient volume 6 - 10 Market share is generally not included unless otherwise stated. Maximum patient volume for sluice References GIPdatabank; CBS Additional remarks In 2015 werden 11 volwassenen behandeld met plerixafor. De verhouding van 1-18 jarigen ten opzichte van 18+ in Nederland in 2017 was 1:3,7. Dit zou betekenen dat er 3 kinderen in aanmerking zouden komen voor deze indicatie-uitbreiding. In het prinses Maxima centrum werden in 2018 6 kinderen behandeld met Mozobil, in 2019 tot op heden 4 kinderen. Expected cost per patient per year Cost € < 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 GIPdatabank; EPAR Additional remarks In 2015 werd er €10.648 vergoed per gebruiker van plerixafor. Aangezien plerixafor per kg lichaamsgewicht wordt toegediend zullen de kosten per patiënt per jaar wellicht iets lager uitvallen. Potential total cost per year Total cost € 88,000.00 Total cost for sluice Additional remarks Off label use Off label use Yes Indications off label use References Additional remarks Indication extension Indication extensions No Indication extensions References Additional remarks Other information Additional remarks