Extended indication Treatment of severe thrombocytopenia in adult patients with chronic liver disease who are scheduled
Therapeutic value Possible equal value
Total cost 1,300,000.00
Registration phase Positive CHMP opinion

Product

Active substance Avatrombopag
Domain Cardiovascular diseases
Reason of inclusion New medicine (specialité)
Main indication Other non-oncological hematological medications
Extended indication Treatment of severe thrombocytopenia in adult patients with chronic liver disease who are scheduled to undergo an invasive procedure
Proprietary name Doptelet
Manufacturer Dova
Mechanism of action Receptor agonist
Route of administration Oral
Therapeutical formulation Capsule
Budgetting framework Intermural (MSZ)
Additional comments Meer informatie over de werking van het product: "small-molecule thrombopoietin receptor (c-Mpl) agonist that stimulates platelet production."

Registration

Registration route Centralised (EMA)
Type of trajectory Normal trajectory
Submission date June 2018
Expected Registration June 2019
Orphan drug No
Registration phase Positive CHMP opinion
Additional comments Study 302: durable platelet responses. Positieve CHMP-opinie april 2019.

Therapeutic value

Current treatment options Eltrombopag
Therapeutic value Possible equal value
Frequency of administration 1 times a day
Dosage per administration 5 mg - 40 mg
References NCT01438840

Expected patient volume per year

Patient volume

100

Market share is generally not included unless otherwise stated.

References GIP-databank, 2016
Additional comments Het aantal personen in Nederland met ITP wordt geschat op ongeveer 5 op de 100.000 mensen (=850 personen). Aantal gebruikers Eltrombopag is 343, terwijl dit voor meer indicaties is geregistreerd dan ITP. Gezien de verwachte gelijke waarde is een realistisch patiëntaantal 100 personen.

Expected cost per patient per year

Cost 13,000.00
Additional comments GIP databank geeft voor Eltrombopag in 2016, €13.139 per patiënt per jaar aan.

Potential total cost per year

Total cost

1,300,000.00

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 Thrombocytopenia - associated with liver disease, pre-surgical procedure
References SPS

Other information

There is currently no futher information available.