Entrectinib Medicine 05 December 2023 Extended indication Two pediatric indications: 1) Rozlytrek as monotherapy for the treatment of paediatric patients with solid tumours that have a ROS1 gene fusion, who have a disease that is locally advanced, metastatic or where surgical resection is likely to result in severe morbidity, and who have not received a prior ROS1 inhibitor, and who have no satisfactory treatment options. 2) indication extension to pediatric patients <12 years with solid tumours that have a NTRK gene fusion who have a disease that is locally advanced, metastatic or where surgical resection is likely to result in severe morbidity, and who have not received a prior NTRK inhibitor, and who have no satisfactory treatment options. Therapeutic value Possible added value Total cost € 577,800.00 Registration phase Registration application pending Product Active substance Entrectinib Domain Oncology Reason of inclusion Indication extension IND Main indication Oncology other Extended indication Two pediatric indications: 1) Rozlytrek as monotherapy for the treatment of paediatric patients with solid tumours that have a ROS1 gene fusion, who have a disease that is locally advanced, metastatic or where surgical resection is likely to result in severe morbidity, and who have not received a prior ROS1 inhibitor, and who have no satisfactory treatment options. 2) indication extension to pediatric patients <12 years with solid tumours that have a NTRK gene fusion who have a disease that is locally advanced, metastatic or where surgical resection is likely to result in severe morbidity, and who have not received a prior NTRK inhibitor, and who have no satisfactory treatment options. Current proprietary name Already available biosimilars / generics Proprietary name Rozlytrek Manufacturer Roche Portfolio holder Roche Mechanism of action Tyrosine kinase inhibitor Route of administration Oral Therapeutical formulation Oral suspension Budgetting framework Intermural (MSZ) Centre of expertise Prinses Maxima Centrum Additional remarks Registration Registration route Centralised (EMA) Type of trajectory Normal trajectory Particularity Unknown ATMP No Submission date April 2023 Expected Registration June 2024 Orphan drug No Registration phase Registration application pending Reimbursement Medicine sluice Additional remarks Therapeutic value Current treatment options Vanwege tumor-agnostisch karakter lastig om huidig opties / standard of care te definiëren. Therapeutic value Possible added value This assessment does not indicate any potential inclusion in the package. Substantiation Er wordt verwacht door experts dat er mogelijke meerwaarde zal zijn van dit medicament. Duration of treatment Median 10.6 month / months Frequency of administration 1 times a day Dosage per administration 600MG References https://pubmed.ncbi.nlm.nih.gov/35395680/ https://www.ema.europa.eu/en/documents/product-information/rozlytrek-epar-product-information_nl.pdf Additional remarks Uit praktische overwegingen hier de dosering voor volwassen aangehouden Expected patient volume per year Patient volume 6 - 12 Market share is generally not included unless otherwise stated. Maximum patient volume for sluice References https://nfk.nl/media/1/Consensus_Rapport_Diagnostiek_en_Behandeling_van_NTRK-Genfusie_Gerelateerde_Solide_Tumoren_14022020.pdf Additional remarks In het consensus rapport over de diagnostiek en behandeling van NTRK-gerelateerde solide tumoren staat benoemd dat er jaarlijks 3-6 kinderen in Nederland geïdentificeerd kunnen worden die in aanmerking komen voor behandeling met een TRK-remmer. Ervan uitgaande dat deze incidentie ook voor kinderen met ROS1-gerelateerde solide tumoren geldt, komen er jaarlijks 6 tot 12 pediatrische patiënten in aanmerking. Expected cost per patient per year Cost € 64,200.00 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 Z-index https://www.ema.europa.eu/en/documents/product-information/rozlytrek-epar-product-information_nl.pdf Additional remarks Uit praktische overwegingen hier de dosering voor volwassen aangehouden (600mg eenmaal daags). Prijs Rozlytrek volgens de Z-index bedraagt €5.350 voor 90 stuks van 200MG. Jaarlijkse kosten bedragen hiermee dus €64.200. Door de dosering van volwassenen te nemen betreft dit wel een overschatting. Potential total cost per year Total cost € 577,800.00 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