Eltrombopag Medicine 07 June 2022 Extended indication Extension of indication to include treatment of adult patients with primary immune thrombocytopenia (ITP) who are refractory to other treatments (e.g. corticosteroids, immunoglobulins) irrespective of time since initial diagnosis Therapeutic value No estimate possible yet Total cost € 5,236,000.00 Registration phase Registration application pending Product Active substance Eltrombopag Domain Hematology Reason of inclusion Indication extension IND Main indication Other hematology Extended indication Extension of indication to include treatment of adult patients with primary immune thrombocytopenia (ITP) who are refractory to other treatments (e.g. corticosteroids, immunoglobulins) irrespective of time since initial diagnosis Current proprietary name Already available biosimilars / generics Proprietary name Revolade Manufacturer Novartis Portfolio holder Mechanism of action Receptor antagonist Route of administration Oral Therapeutical formulation Tablet Budgetting framework Extramural (GVS) Centre of expertise Additional remarks Registration Registration route Centralised (EMA) Type of trajectory Unknown Particularity Unknown ATMP Unknown Submission date January 2022 Expected Registration November 2022 Orphan drug No Registration phase Registration application pending Reimbursement Medicine sluice Additional remarks Therapeutic value Current treatment options Therapeutic value No estimate possible yet 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 References NCT03524612 (TAPER) Additional remarks Participants will be treated with eltrombopag to induce sustained remission to reach a target platelet count of ≥ 100×109/L (CR), after 1st line steroids have failed. eltrombopag is for oral use and comes in 12.5, 25, 50 and 75 mg tablets; prescribed dose is taken once daily. Expected patient volume per year Patient volume < 680 Market share is generally not included unless otherwise stated. Maximum patient volume for sluice References erfelijkheid.nl Additional remarks ITP komt bij ongeveer 1 tot 4 op de 100.000 mensen voor. Expected cost per patient per year Cost € 1,400.00 - 14,000.00 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 In 2021 werd er per gebruiker €14.025 vergoed. Potential total cost per year Total cost € 5,236,000.00 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