Hydrocortison Medicine 08 December 2020 Extended indication Treatment of congenital adrenal hyperplasia (CAH) in adolescents aged 12 years and over and adults. Therapeutic value No estimate possible yet Total cost € 8,750,000.00 Registration phase Registered and reimbursed Product Active substance Hydrocortison Domain Metabolism and Endocrinology Reason of inclusion New medicine (specialité) Main indication Metabolic diseases Extended indication Treatment of congenital adrenal hyperplasia (CAH) in adolescents aged 12 years and over and adults. Current proprietary name Already available biosimilars / generics Proprietary name Efmody Manufacturer Diurnal Portfolio holder Mechanism of action Receptor agonist Route of administration Oral Therapeutical formulation Capsule Budgetting framework Extramural (GVS) Centre of expertise Additional remarks Hydrocortisone extended release glucocorticoid receptor agonist. Registration Registration route Centralised (EMA) Type of trajectory Normal trajectory Particularity New therapeutical formulation ATMP Unknown Submission date March 2020 Expected Registration June 2021 Orphan drug Yes Registration phase Registered and reimbursed Reimbursement Medicine sluice Additional remarks Positieve CHMP-opinie in maart 2021. Therapeutic value Current treatment options Standaard glucocorticoid therapie: hydrocortisone, prednisone, prednisolone, dexamethasone. Therapeutic value No estimate possible yet This assessment does not indicate any potential inclusion in the package. Substantiation Het voordeel van Chronocort ten opzichte van Plenadren is de mooiere release en langdurige werking van het geneesmiddel. Duration of treatment Average 0 continuous Frequency of administration 2 times a day Dosage per administration 5mg, 10mg, or 20mg References NCT03062280; NCT02716818 Additional remarks Therapeutic value: Chronocort provides improved biochemical disease control by providing early morning exposure to cortisol, thereby controlling the otherwise observed early morning adrenal androgen excess in CAH, with reduction in steroid dose over time and patient-reported benefits. Duration of treatment: CAH is a lifelong disorder, classic CAH patients need to take medication daily for their entire lives. Expected patient volume per year Patient volume 1,500 - 2,000 Market share is generally not included unless otherwise stated. Maximum patient volume for sluice References ntvg; Additional remarks Het totale aantal patiënten met bijnierinsufficiëntie ligt vermoedelijk tussen de 2.000-4.000. Er wordt verwacht dat er substitutie van plenadren plaats zal vinden en nieuwe patiënten gebruik zullen maken van hydrocortison. Dit zal leiden tot een mogelijk patiëntvolume tussen de 1.500-2.000 patiënten. Expected cost per patient per year Cost € 5,000.00 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 https://www.hardmanandco.com/wp-content/uploads/2020/02/Diurnal-Interim-update-28-Feb-2020.pdf Additional remarks Krijgt een benchmark prijs van $6,000 per patiënt per jaar (ongeveer €5.000) Potential total cost per year Total cost € 8,750,000.00 Total cost for sluice Additional remarks Off label use Off label use Unknown Indications off label use References Additional remarks Indication extension Indication extensions Yes Indication extensions Adrenal insufficiency References NCT01735617 Additional remarks Indien chronocort ook voor bijnierinsufficiëntie ingezet gaat worden zal het patiëntvolume vele malen hoger zijn. Other information Additional remarks