Rurioctocog alfa pegol

Extended indication	Haemophilia A; severe in adults and children from 0 to 75 years, Second or subsequent line, for treatment and prevention of bleeding, routine prophylaxis and perioperative bleeding.
Therapeutic value	No judgement
Total cost	164,937,500.00

Product

Active substance	Rurioctocog alfa pegol
Domain	Oncology and Hematology
Main indication	Haemophilia
Extended indication	Haemophilia A; severe in adults and children from 0 to 75 years, Second or subsequent line, for treatment and prevention of bleeding, routine prophylaxis and perioperative bleeding.
Proprietary name	Adynovi
Manufacturer	Shire
Route of administration	Intravenous
Therapeutical formulation	Injection
Budgetting framework	Intermural (MSZ)
Additional remarks	PEGylated versie van Advate.

Registration

Registration route	Centralised (EMA)
Type of trajectory	Normal trajectory
Submission date	April 2016
Expected Registration	January 2018
Orphan drug	No
Additional remarks	Positieve CHMP opinie november 2017.

Therapeutic value

Current treatment options	Verschillende recombinante factor VIII eiwitten.
Therapeutic value	No judgement
Duration of treatment	continuous
Frequency of administration	3 times a week
Dosage per administration	20-40 IE/kg
References	Farmacotherapeutisch kompas
Additional remarks	Toediningsfrequentie: 1 maal per 2-3 dagen; Obv langetermijnprofylaxe beschreven in FK.

Expected patient volume per year

Patient volume	< 725 Market share is generally not included unless otherwise stated.
Additional remarks	Max. patiënten: Ongeveer 1.450 patiënten hebben hemofilie A. De helft hiervan heeft een ziektestaat die moderate/severe is en wordt behandeld met factor VIII. Realistische patientenaantallen lastig in te schatten.

Patient volume

< 725

Market share is generally not included unless otherwise stated.

Additional remarks

Max. patiënten: Ongeveer 1.450 patiënten hebben hemofilie A. De helft hiervan heeft een ziektestaat die moderate/severe is en wordt behandeld met factor VIII. Realistische patientenaantallen lastig in te schatten.

Expected cost per patient per year

Cost	110,000.00 - 345,000.00
References	Medicijnkosten.nl; NVHP; Fabrikant
Additional remarks	Berekend obv de prijs voor octocog alfa via medicijnkosten.nl en behandelschema beschreven in het farmacotherapeutisch kompas voor een patient van 70 kg langetermijnprofilaxe: €170.000-520.000. De nederlands vereniging voor hemofiliepatiënten geeft aan dat alle hemofiliebehandelingen in 2014 in totaal €135 miljoen kostten (voor 1600 hemofiliepatienten). Fabrikant (nov 2017): 110.000-345.000 p.p.p.j.

Potential total cost per year

Total cost	164,937,500.00 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

164,937,500.00

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

There is currently nothing known about indication extensions.

Other information

There is currently no futher information available.