Bedaquiline Medicine 08 June 2021 Extended indication Sirturo is indicated for use as part of an appropriate combination regimen for pulmonary multidrug‑resistant tuberculosis (MDR‑TB) in adults and paediatric patients (5 years to less than 18 years of age and weighing at least 15 kg) when an effective treatment regimen cannot otherwise be composed for reasons of resistance or tolerability. Therapeutic value Possible added value Total cost 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 paediatric patients (5 years to less than 18 years of age and weighing at least 15 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 New therapeutical formulation ATMP No Submission date November 2019 Expected Registration March 2021 Orphan drug Yes Registration phase Registered and reimbursed Reimbursement Medicine sluice Additional remarks Positieve CHMP-opinie in januari 2021. Therapeutic value Current treatment options Geen vergelijkende medicamenten beschikbaar. 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 een klein aantal kinderen een meerwaarde kan hebben. Duration of treatment Frequency of administration Dosage per administration References Additional remarks Extra sterkte en aanvullende pediatrische formulering. Expected patient volume per year Patient volume 1 - 2 Market share is generally not included unless otherwise stated. Maximum patient volume for sluice References Surveillance rapport RIVM MDR/XDR TB; https://www.rivm.nl/tuberculose Additional remarks Het totaal aantal patiënten met TBC in 2020 betrof 623, hiervan waren er 9 MRD. Het aantal kinderen hierin zal erg klein zijn. De getallen bevinden zich in een dalende trend. Er worden de komende jaren 1-2 patiënten verwacht die in aanmerking komen. Expected cost per patient per year Cost References medicijnkosten.nl Additional remarks De prijs voor 20mg tabletten nog niet beschikbaar. De prijs voor 100mg betreft nu €116,41 inclusief btw en €106,80 exclusief btw. Potential total cost per year Total cost Total cost for sluice Additional remarks limited. Verwachting is zelfde range per jaar als voor adult/adolescent indicatie Off label use Off label use Unknown Indications off label use References Additional remarks Indication extension Indication extensions No Indication extensions References Adis Insight Additional remarks Geen andere lopende fase 3 studies binnen een andere indicatie. Other information Additional remarks