Treprostinil

Extended indication	Treatment of adults with chronic thromboembolic pulmonary hypertension (CTEPH) who cannot have surgery, and in patients whose CTEPH continues or has come back after surgery.
Therapeutic value	No judgement
Total cost	4,200,000.00
Registration phase	Registration application pending

Product

Active substance	Treprostinil
Domain	Cardiovascular diseases
Reason of inclusion	New medicine (specialité)
Main indication	Other medication for cardiovascular diseases
Extended indication	Treatment of adults with chronic thromboembolic pulmonary hypertension (CTEPH) who cannot have surgery, and in patients whose CTEPH continues or has come back after surgery.
Proprietary name	Treprostinil SciPharm Sàrl
Manufacturer	SciPharm Sàrl
Mechanism of action	Vasodilator
Route of administration	Oral
Therapeutical formulation	Tablet
Budgetting framework	Extramural (GVS)
Additional remarks	Treprostinil diethanolamide is een vasodilator, synthetische analoog van prostacycline.

Registration

Registration route	Centralised (EMA)
Type of trajectory	Normal trajectory
Particularity	New therapeutical formulation
Submission date	March 2019
Expected Registration	April 2020
Orphan drug	Yes
Registration phase	Registration application pending
Additional remarks	De fabrikant heeft een eerdere registratieaanvraag in september 2018 teruggetrokken omdat het niet kon voldoen aan de vraag van de EMA om additionele data aan te leveren.

Therapeutic value

Current treatment options	Treprostinil IV (Remodulin)
Therapeutic value	No judgement
Substantiation	Treprostinil zal naar verwachting niet veel voorgeschreven worden. Het gebruik zal zich beperken tot tertiaire centra.
References	United Therapeutics
Additional remarks	De FREEDOM-EV trial heeft de primaire eindpunten gehaald.

Expected patient volume per year

Patient volume	< 28 Market share is generally not included unless otherwise stated.
References	GIPdatabank
Additional remarks	In 2016 waren er 28 gebruikers van Remodulin.

Patient volume

< 28

Market share is generally not included unless otherwise stated.

References

GIPdatabank

Additional remarks

In 2016 waren er 28 gebruikers van Remodulin.

Expected cost per patient per year

Cost	150,000.00
References	GIPdatabank
Additional remarks	In 2016 werd er €150.720 vergoed per gebruiker.

Potential total cost per year

Total cost	4,200,000.00 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

4,200,000.00

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	Systemic sclerosis
References	EMA; Clinicaltrials.gov
Additional remarks	Orphan designation voor deze indicatie, momenteel bevinden studies zich in fase II.

Other information

There is currently no futher information available.