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Hydrocortison

Efmody

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

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.
Proprietary name Efmody
Manufacturer Diurnal
Mechanism of action Receptor agonist
Route of administration Oral
Therapeutical formulation Capsule
Budgetting framework Extramural (GVS)
Additional remarks Hydrocortisone extended release glucocorticoid receptor agonist.

Registration

Registration route Centralised (EMA)
Type of trajectory Normal trajectory
Particularity New therapeutical formulation
Submission date March 2020
Expected Registration June 2021
Orphan drug Yes
Registration phase Registered
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
Substantiation Het voordeel van Chronocort ten opzichte van Plenadren is de mooiere release en langdurige werking van het geneesmiddel.
Duration of treatment 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.
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
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

8,750,000.00

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.

Off label use

There is currently nothing known about off label use.

Indication extension

Indication extension 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

There is currently no futher information available.