Hydrocortison

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.

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 (ongeveer €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.

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.