Avatrombopag Medicine 12 June 2018 Extended indication Treatment of severe thrombocytopenia in adult patients with chronic liver disease who are scheduled to undergo an invasive procedure. Therapeutic value No estimate possible yet Total cost € 1,300,000.00 Registration phase Registered and reimbursed Product Active substance Avatrombopag Domain Cardiovascular diseases Reason of inclusion New medicine (specialité) Main indication Other non-oncological hematological medications Extended indication Treatment of severe thrombocytopenia in adult patients with chronic liver disease who are scheduled to undergo an invasive procedure. Current proprietary name Already available biosimilars / generics Proprietary name Doptelet Manufacturer Sobi Portfolio holder Mechanism of action Receptor agonist Route of administration Oral Therapeutical formulation Capsule Budgetting framework Extramural (GVS) Centre of expertise Additional remarks Meer informatie over de werking van het product: "small-molecule thrombopoietin receptor (c-Mpl) agonist that stimulates platelet production." Registration Registration route Centralised (EMA) Type of trajectory Normal trajectory Particularity Unknown ATMP Unknown Submission date June 2018 Expected Registration June 2019 Orphan drug No Registration phase Registered and reimbursed Reimbursement Medicine sluice Additional remarks Study 302: durable platelet responses. Positieve CHMP-opinie april 2019. Geregistreerd in juni 2019. GVS-advies gepubliceerd in juni 2021. Therapeutic value Current treatment options Op dit moment enkel platelet transfusie, in de toekomst ook wellicht lusutrombopag. Therapeutic value No estimate possible yet This assessment does not indicate any potential inclusion in the package. Substantiation Duration of treatment Average 5 day / days Frequency of administration 1 times a day Dosage per administration 40 of 60 mg References NCT01438840 Additional remarks Dosering is afhankelijk van platelet count Expected patient volume per year Patient volume 100 Market share is generally not included unless otherwise stated. Maximum patient volume for sluice References GIP-databank, 2016 Additional remarks Het aantal personen in Nederland met ITP wordt geschat op ongeveer 5 op de 100.000 mensen (=850 personen). Aantal gebruikers eltrombopag is 343, terwijl dit voor meer indicaties is geregistreerd dan ITP. Gezien de verwachte gelijke waarde is een realistisch patiëntaantal 100 personen. Expected cost per patient per year Cost € 13,000.00 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 fabrikant Additional remarks GIP databank geeft voor eltrombopag in 2016, €13.139 per patiënt per jaar aan. Dit is echter een inschatting. Kosten per patiënt van eltrombopag gaat om een niet helemaal vergelijkbare patiëntenpopulatie. Wat betreft ITP: Zoals in de huidige Horizonscan Geneesmiddelen ook al is aangegeven, is eltrombopag voor meerdere indicaties geregistreerd. Voor Doptelet zal het waarschijnlijk alleen om volwassen patiënten met ITP gaan (voor eltrombopag ook om kinderen). Potential total cost per year Total cost € 1,300,000.00 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 Thrombocytopenia - associated with liver disease, pre-surgical procedure References SPS Additional remarks Other information Additional remarks