Riociguat Medicine 07 December 2022 Extended indication Adempas is indicated for the treatment of PAH in paediatric patients aged less than 18 years of age and body weight ≥ 50 kg with WHO Functional Class (FC) II to III in combination with endothelin receptor antagonists. Therapeutic value No estimate possible yet Total cost € 240,000.00 Registration phase Registered and reimbursed Product Active substance Riociguat Domain Lung diseases Reason of inclusion Indication extension IND Main indication Lung diseases other Extended indication Adempas is indicated for the treatment of PAH in paediatric patients aged less than 18 years of age and body weight ≥ 50 kg with WHO Functional Class (FC) II to III in combination with endothelin receptor antagonists. Current proprietary name Already available biosimilars / generics Proprietary name Adempas Manufacturer MSD Portfolio holder MSD Mechanism of action Vasodilator Route of administration Oral Therapeutical formulation Coated tablet Budgetting framework Extramural (GVS) Centre of expertise Additional remarks Registration Registration route Centralised (EMA) Type of trajectory Normal trajectory Particularity Unknown ATMP No Submission date September 2022 Expected Registration May 2023 Orphan drug Yes Registration phase Registered and reimbursed Reimbursement Medicine sluice Additional remarks Positieve opinie CHMPO in april 2023. Therapeutic value Current treatment options Therapeutic value No estimate possible yet This assessment does not indicate any potential inclusion in the package. Substantiation Het veiligheidsprofiel van riociguat lijkt acceptabel te zijn aan de hand van de studieresultaten. Duration of treatment Frequency of administration Dosage per administration References The PATENT–CHILD study. Pulm Circ. 2022 Jul; 12(3); Additional remarks Expected patient volume per year Patient volume < 10 Market share is generally not included unless otherwise stated. Maximum patient volume for sluice References Ntvg.nl (1); Gorenflo. Cardiovasc Diagn Ther. 2021 (2); Expertopinie (3); Additional remarks De prevalentie van PAH in Nederland is ongeveer 15-52 per 1.000.000 inwoners. Bij kinderen is het echter vele malen zeldzamer met een incidentie van 0,7 (iPAH) en 2,2 (PAH-AHF) per miljoen kinderen (2). Er zijn ongeveer 2,5 miljoen kinderen in deze leeftijdsgroep in Nederland. Er wordt verwacht dat er niet meer dan 10 patiënten in aanmerking zullen komen (3). Expected cost per patient per year Cost € < 24,000.00 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 Voor de huidige indicatie werd in 2021 €24.202 per gebruiker vergoed. Potential total cost per year Total cost € 240,000.00 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