Extended indication

Replacement therapy for congenital adrenal hyperplasia (CAH) in adolescents aged 12 years and over a

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.

Proprietary name

Chronocort

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

February 2021

Orphan drug

Yes

Registration phase

Registration application pending

Therapeutic value

Current treatment options

Standaard glucocorticoid therapie: hydrocortisone, prednisone, prednisolone, dexamethasone.

Therapeutic value

No judgement

Substantiation

Het voordeel van Chronocort ten opzichte van Plenadren is de mooiere release en langdurige werking van het geneesmiddel.

Frequency of administration

1 times a day

Dosage per administration

5mg, 10mg of 20mg

References
NCT03062280; NCT02716818

Expected patient volume per year

Patient volume

468

Market share is generally not included unless otherwise stated.

References
ntvg;
Additional comments
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

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 (~€5.000)

Potential total cost per year

Total cost

2,340,000.00

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.