Natriumoxybaat

Extended indication	Narcolepsy with cataplexy in children (ages 7-17)
Therapeutic value	No judgement
Total cost	480,000.00
Registration phase	Clinical trials

Product

Active substance	Natriumoxybaat
Domain	Neurological disorders
Reason of inclusion	Indication extension
Main indication	Sleep disorders
Extended indication	Narcolepsy with cataplexy in children (ages 7-17)
Proprietary name	Xyrem
Manufacturer	UCB
Route of administration	Oral
Therapeutical formulation	Drink
Budgetting framework	Extramural (GVS)
Additional remarks	Central-nervous-system depressant.

Registration

Registration route	Centralised (EMA)
Submission date	2019
Expected Registration	2020
Orphan drug	No
Registration phase	Clinical trials
Additional remarks	In juni 2018 presenteerde de fabrikant positieve langetermijnresultaten van de EXPRESS studie.

Therapeutic value

Therapeutic value	No judgement
Duration of treatment	continuous
Frequency of administration	1 times a day
Dosage per administration	≤9 g/nacht
References	Farmacotherapeutisch kompas; NCT02221869

Expected patient volume per year

Patient volume	< 60 Market share is generally not included unless otherwise stated.
Additional remarks	In 2016 gebruikten 406 mensen Xyrem. In 2017 waren er 2.162.869 inwoners in Nederland van de leeftijd 7-17 en 13.677.409 van de leeftijd >17. Als de populatie >17.406 gebruikers telde dan zou dit voor 7-17 betekenen dat er 64 gebruikers zijn.

Patient volume

< 60

Market share is generally not included unless otherwise stated.

Additional remarks

In 2016 gebruikten 406 mensen Xyrem. In 2017 waren er 2.162.869 inwoners in Nederland van de leeftijd 7-17 en 13.677.409 van de leeftijd >17. Als de populatie >17.406 gebruikers telde dan zou dit voor 7-17 betekenen dat er 64 gebruikers zijn.

Expected cost per patient per year

Cost	8,000.00
References	GIPdatabank
Additional remarks	In 2016 werd €8.108 vergoed per gebruiker van Xyrem.

Potential total cost per year

Total cost	480,000.00 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

480,000.00

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	Fibromyalgia
References	Clinicaltrials.gov

Other information

There is currently no futher information available.