Macitentan

Extended indication	Extension of Indication to include treatment of patients with inoperable chronic thromboembolic pulmonary hypertension (CTEPH)
Therapeutic value	No judgement
Total cost	4,320,000.00
Registration phase	Registration application pending

Product

Active substance	Macitentan
Domain	Cardiovascular diseases
Reason of inclusion	Indication extension
Main indication	Antithrombotic medications
Extended indication	Extension of Indication to include treatment of patients with inoperable chronic thromboembolic pulmonary hypertension (CTEPH)
Proprietary name	Opsumit
Manufacturer	Actelion
Mechanism of action	Receptor antagonist
Route of administration	Oral
Therapeutical formulation	Tablet
Budgetting framework	Extramural (GVS)
Additional remarks	Endothelin receptor antagonist.

Registration

Registration route	Centralised (EMA)
Type of trajectory	Normal trajectory
Submission date	September 2018
Expected Registration	August 2019
Orphan drug	No
Registration phase	Registration application pending
Additional remarks	Orphan drug status in US for pulmonary hypertension

Therapeutic value

Current treatment options	Riociguat
Therapeutic value	No judgement
Substantiation	De verwachting is dat er substitutie van riociguat zal plaatsvinden.
Duration of treatment	continuous
Frequency of administration	1 times a day
Dosage per administration	10 mg
References	NCT02060721, NCT02021292, NCT02565030

Expected patient volume per year

Patient volume	< 108 Market share is generally not included unless otherwise stated.
References	GIPdatabank
Additional remarks	In 2017 werden 108 mensen behandeld met riociguat (Adempas). Aangezien de verwachting is dat er substitutie zal plaatsvinden is dit het maximale aantal patiënten wat in aanmerking zal komen voor deze behandeling.

Patient volume

< 108

Market share is generally not included unless otherwise stated.

References

GIPdatabank

Additional remarks

In 2017 werden 108 mensen behandeld met riociguat (Adempas). Aangezien de verwachting is dat er substitutie zal plaatsvinden is dit het maximale aantal patiënten wat in aanmerking zal komen voor deze behandeling.

Expected cost per patient per year

Cost	40,000.00
References	Medicijnkosten.nl
Additional remarks	De prijs voor een tablet van 10 mg is €109,88. Uitgaande van een behandelduur van een jaar komt dit neer op ongeveer €40.000.

Potential total cost per year

Total cost	4,320,000.00 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

4,320,000.00

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	Fase 3: Congenital heart defects; Eisenmenger complex
References	Adis Insight

Other information

There is currently no futher information available.