Selinexor Medicine 12 June 2019 Extended indication Patients with relapsed or refractory multiple myeloma who have received at least three prior lines of therapy and whose disease is refractory to at least one PI, one IMiD, and one anti-CD38 monoclonal antibody, and to their most recent treatment regimen. Therapeutic value No judgement Total cost € 6,640,000.00 Registration phase Registration application pending Product Active substance Selinexor Domain Oncology and Hematology Reason of inclusion New medicine (specialité) Main indication Multiple Myeloma Extended indication Patients with relapsed or refractory multiple myeloma who have received at least three prior lines of therapy and whose disease is refractory to at least one PI, one IMiD, and one anti-CD38 monoclonal antibody, and to their most recent treatment regimen. Current proprietary name Already available biosimilars / generics Proprietary name Manufacturer Karyopharm Portfolio holder Mechanism of action Other Route of administration Oral Therapeutical formulation Unknown Budgetting framework Intermural (MSZ) Centre of expertise Additional remarks CRM1 nuclear export inhibitor Registration Registration route Centralised (EMA) Type of trajectory Normal trajectory Particularity Unknown ATMP Unknown Submission date February 2019 Expected Registration March 2020 Orphan drug Yes Registration phase Registration application pending Reimbursement Medicine sluice Additional remarks Werd eerder beoordeeld via accelerated assessment, maar dat is gewijzigd per juni 2019. Therapeutic value Current treatment options Therapeutic value No judgement This assessment does not indicate any potential inclusion in the package. Substantiation Duration of treatment Median 9 week / weeks Frequency of administration 2 times a week Dosage per administration 80 mg References NCT02336815; Chari et al. N Engl J Med 2019; 381:727-738 Additional remarks Expected patient volume per year Patient volume < 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 maximaal 166 patiënten. De inschatting is dat dit aantal nog lager zal liggen gegeven de vele behandelopties. Expected cost per patient per year Cost € 40,000.00 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 https://www.reuters.com/article/us-karyopharm-fda-idUSKCN1TY2FI Additional remarks In de VS is de lijstprijs per maand bepaald op $22.000 (~€20.000). De mediane behandelduur is 9 weken wat neerkomt op ongeveer €40.000. Potential total cost per year Total cost € 6,640,000.00 Total cost for sluice Additional remarks Off label use Off label use Unknown Indications off label use References Additional remarks Indication extension Indication extensions Yes Indication extensions Liposarcoma; Endometrial cancer; Acute myeloid leukaemia; Breast cancer; Cervical cancer; Cutaneous T-cell lymphoma; Diffuse large B cell lymphoma; Glioblastoma; Myelodysplastic syndromes; Neuroendocrine tumours; Ovarian cancer; Peripheral T-cell lymphoma; Prostate cancer; Small cell lung cancer; Squamous cell cancer; Thymoma; Chronic myelomonocytic leukaemia; Non-Hodgkin's lymphoma; Non-small cell lung cancer; Soft tissue sarcoma References Clinicaltrials.gov Additional remarks Other information Additional remarks