Bedaquiline Medicine 12 June 2019 Extended indication Sirturo is indicated for use as part of an appropriate combination regimen for pulmonary multidrug‑resistant tuberculosis (MDR‑TB) in adults and adolescent patients (12 years to less than 18 years of age and weighing at least 30 kg) when an effective treatment regimen cannot otherwise be composed for reasons of resistance or tolerability. Therapeutic value Possible added value Total cost € 43,468.00 Registration phase Registered and reimbursed Product Active substance Bedaquiline Domain Infectious diseases Reason of inclusion Indication extension IND Main indication Bacterial infections Extended indication Sirturo is indicated for use as part of an appropriate combination regimen for pulmonary multidrug‑resistant tuberculosis (MDR‑TB) in adults and adolescent patients (12 years to less than 18 years of age and weighing at least 30 kg) when an effective treatment regimen cannot otherwise be composed for reasons of resistance or tolerability. Current proprietary name Already available biosimilars / generics Proprietary name Sirturo Manufacturer Janssen Portfolio holder Mechanism of action Enzyme inhibitor Route of administration Oral Therapeutical formulation Tablet Budgetting framework Intermural (MSZ) Centre of expertise Additional remarks ATP synthase-remmer Registration Registration route Centralised (EMA) Type of trajectory Normal trajectory Particularity Unknown ATMP Unknown Submission date November 2018 Expected Registration February 2020 Orphan drug No Registration phase Registered and reimbursed Reimbursement Medicine sluice Additional remarks Positieve CHMP-opinie in december 2019. Geregistreerd in januari 2020. Therapeutic value Current treatment options Therapeutic value Possible added value This assessment does not indicate any potential inclusion in the package. Substantiation Bij volwassenen behoort bedaquiline al tot de standaardzorg. Er wordt verwacht dat het geneesmiddel ook bij adolescenten een meerwaarde zal hebben. Duration of treatment Average 24 week / weeks Frequency of administration 1 times a day Dosage per administration 200-400 mg References Additional remarks De eerste twee weken ontvangt de patiënt 400mg eenmaal per dag, daarna 200mg driemaal per week. Expected patient volume per year Patient volume 1 - 3 Market share is generally not included unless otherwise stated. Maximum patient volume for sluice References Surveillance rapport RIVM geeft indicatie bij MDR / XDR TB. Additional remarks Circa 5% van de TB patiënten zijn kinderen. Indicatie hoogstens 5 maal per jaar voor totale cohort (maximaal 1 kind per jaar). Recente WHO richtlijn wordt bedaquiline als belangrijkste aangemerkt gezien ontraden van injectables. In toekomst maximaal 15 per jaar geschat voor het totale cohort (dus maximaal 2-3 kinderen per jaar). Expected cost per patient per year Cost € 21,734.00 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 G- standaard; SmPC; fabrikant Additional remarks De huidige lijstprijs van 100mg, 188 stuks is € 21.734,61 exclusief BTW. Potential total cost per year Total cost € 43,468.00 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