Pirfenidone Medicine 07 June 2022 Extended indication Extension of indication to include treatment of ‘advanced’ idiopathic pulmonary fibrosis (IPF) by the deletion of the current qualifier ‘mild to moderate’, in IPF patients with advanced lung function impairment (DLco < 40% of predicted) and at high risk of grade 3 pulmonary hypertension. Therapeutic value No estimate possible yet Total cost € 1,050,000.00 Registration phase Registered Product Active substance Pirfenidone Domain Lung diseases Reason of inclusion Indication extension IND Main indication Lung diseases other Extended indication Extension of indication to include treatment of ‘advanced’ idiopathic pulmonary fibrosis (IPF) by the deletion of the current qualifier ‘mild to moderate’, in IPF patients with advanced lung function impairment (DLco < 40% of predicted) and at high risk of grade 3 pulmonary hypertension. Current proprietary name Already available biosimilars / generics Proprietary name Esbriet Manufacturer Roche Portfolio holder Roche Mechanism of action Unknown Route of administration Oral Therapeutical formulation Tablet Budgetting framework Extramural (GVS) Centre of expertise Additional remarks Collagen inhibitor; Cytokine inhibitor Registration Registration route Centralised (EMA) Type of trajectory Normal trajectory Particularity Unknown ATMP No Submission date February 2022 Expected Registration March 2023 Orphan drug No Registration phase Registered Reimbursement Medicine sluice Additional remarks M.T. Durheim. Outcomes of patients with advanced idiopathic pulmonary fibrosis. Positieve CHMP opinie februari 2023. Treated with nintedanib or pirfenidone in a real-world multicentre cohort. Respirology. 2021; Weesstatus is verlopen. Therapeutic value Current treatment options Nintedanib Therapeutic value No estimate possible yet This assessment does not indicate any potential inclusion in the package. Substantiation Van de 502 patiënten in het onderzoek hadden er 66 (13%) geavanceerd IPF. Jaarlijkse verandering in FVC was125ml (95% CI 163, 87) bij patiënten met een milde vorm van IPF en +28ml (95% CI 96, +152) onder patiënten met geavanceerde IPF. De therapeutische waarde of wel remmend effect of achteruitgang longfunctie lijkt daarmee niet minder dan in niet-advanced patiënten. Duration of treatment Frequency of administration Dosage per administration References Durheim et al., (2021) Outcomes of patients with advanced idiopathic pulmonary fibrosis treated with nintedanib or pirfenidone in a real-world multicentre cohort. Respirology. 2021 Oct;26(10):982-988 (1) Additional remarks Expected patient volume per year Patient volume 60 Market share is generally not included unless otherwise stated. Maximum patient volume for sluice References Jaarverslag Longfibrosepatiëntenvereniging 2017 (1); Expert opinie (2); GIP-databank (3); Additional remarks Er zijn ongeveer 3.400 longfibrose patiënten in Nederland 60% daarvan heeft IPF. Dit komt neer op 2.040 IPF patiënten. Ongeveer 10% heeft de classificatie 'advanced IPF', dit zijn 200 patiënten(2). Voor licht tot matige IPF zijn er 611 gebruikers van pirfenidon in 2020 bekend (3). Naar schatting zullen ongeveer 60 patiënten extra per jaar in aanmerking komen voor pirfenidon (2). Expected cost per patient per year Cost € 17,000.00 - 18,000.00 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 In 2020 werd er per patiënt €17.327 vergoed aan pirfenidon. Potential total cost per year Total cost € 1,050,000.00 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