Hydrocortisone Medicine 08 December 2020 Extended indication Replacement therapy for congenital adrenal hyperplasia (CAH) in adolescents aged 12 years and over and adults. Therapeutic value No judgement Total cost € 2,340,000.00 Registration phase Registration application pending Product Active substance Hydrocortisone Domain Metabolism and Endocrinology Reason of inclusion New medicine (specialité) Main indication Metabolic diseases Extended indication Replacement therapy for congenital adrenal hyperplasia (CAH) in adolescents aged 12 years and over and adults. Current proprietary name Already available biosimilars / generics Proprietary name Chronocort 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 February 2021 Orphan drug Yes Registration phase Registration application pending Reimbursement Medicine sluice Additional remarks Therapeutic value Current treatment options Standaard glucocorticoid therapie: hydrocortisone, prednisone, prednisolone, dexamethasone. Therapeutic value No judgement 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 Frequency of administration 1 times a day Dosage per administration 5mg, 10mg of 20mg References NCT03062280; NCT02716818 Additional remarks Expected patient volume per year Patient volume 468 Market share is generally not included unless otherwise stated. Maximum patient volume for sluice References ntvg; Additional remarks De prevalentie van de klassieke vorm van AGS is ongeveer 1:12.000 pasgeborenen. Dit betekent dat er in totaal 1.417 patiënten in Nederland zijn. De verwachting is dat ongeveer 1/3 deel van de patiënten over zal stappen op Chronocort. Dit zal leiden tot een mogelijk patiëntvolume van 468 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 price van $6,000 per patiënt per jaar (~€5.000) Potential total cost per year Total cost € 2,340,000.00 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