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 comments
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 comments
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 comments
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 comments
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 comments
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.

Off label use

There is currently nothing known about off label use.

Indication extension

Indication extension

Yes

Indication extensions

Adrenal insufficiency

References
NCT01735617
Additional comments
Indien chronocort ook voor bijnierinsufficiëntie ingezet gaat worden zal het patiëntvolume vele malen hoger zijn.

Other information

There is currently no futher information available.