Pacritinib Medicine 12 June 2018 Extended indication Patients with myelofibrosis who have thrombocytopenia Therapeutic value No judgement Total cost € 945,000.00 Registration phase Registration application pending Product Active substance Pacritinib Domain Oncology and Hematology Reason of inclusion Main indication Other hematology Extended indication Patients with myelofibrosis who have thrombocytopenia Current proprietary name Already available biosimilars / generics Proprietary name Enpaxiq Manufacturer Cti Portfolio holder Mechanism of action Unknown Route of administration Oral Therapeutical formulation Capsule Budgetting framework Intermural (MSZ) Centre of expertise Additional remarks Tyrosine kinase inhibitor gericht tegen JAK2 en FLT3 Registration Registration route Centralised (EMA) Type of trajectory Normal trajectory Particularity New medicine ATMP Unknown Submission date July 2017 Expected Registration August 2018 Orphan drug Yes Registration phase Registration application pending Reimbursement Medicine sluice Additional remarks Pacritinib was al eerder ingediend in juni 2016 maar werd teruggetrokken in maart 2017 vanwege tijdtekort om nieuwe data in de aanvraag te includeren. Therapeutic value Current treatment options Ruxolitinib Therapeutic value No judgement This assessment does not indicate any potential inclusion in the package. Substantiation Duration of treatment Median 15.6 month / months Frequency of administration 1 times a day Dosage per administration 400 mg References Additional remarks Expected patient volume per year Patient volume 21 - 33 Market share is generally not included unless otherwise stated. Maximum patient volume for sluice References NKR; Mesa et al. Lancet Haematol. 2017 May;4(5):e225-e236. Additional remarks 118 diagnoses in 2016. Trombocytopenie komt voor bij 18-28% van de myelofibrosis patiënten (21-33 patiënten). Expected cost per patient per year Cost € 30,000.00 - 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 GIPdatabank Additional remarks De vergoeding per patiënt in 2015 voor de huidige behandelmethode met Ruxolitinib was €32.174. Potential total cost per year Total cost € 945,000.00 Total cost for sluice Additional remarks Off label use Off label use Unknown Indications off label use References Additional remarks Indication extension Indication extensions Unknown Indication extensions References Additional remarks Other information Additional remarks