Extended indication Transthyretin cardiomyopathy
Therapeutic value Possible added value
Total cost 250,500,000.00
Registration phase Clinical trials

Product

Active substance Tafamidis
Domain Cardiovascular diseases
Reason of inclusion Indication extension
Main indication Other medication for cardiovascular diseases
Extended indication Transthyretin cardiomyopathy
Proprietary name Vyndaqel
Manufacturer Pfizer
Route of administration Oral
Therapeutical formulation Capsule, soft
Budgetting framework Extramural (GVS)
Additional remarks Tafamidis is a kinetic stabilizer of TTR that inhibits misfolding and amyloid formation.

Registration

Registration route Centralised (EMA)
Expected Registration November 2019
Orphan drug Yes
Registration phase Clinical trials
Additional remarks Scrip: "ATTR-ACT; met primary endpoint". De fabrikant verwacht registratie in Q4 2019.

Therapeutic value

Current treatment options geen
Therapeutic value Possible added value
Substantiation effect op sterfte en events
Frequency of administration 1 times a day
Dosage per administration 20 mg / 80 mg

Expected patient volume per year

Patient volume

10 - 5,000

Market share is generally not included unless otherwise stated.

References https://www.pfizer.com/files/news/TTR-CM-Disease-Backgrounder.pdf
Additional remarks Aantallen zijn lastig in te schatten. Pfizer: "It is currently estimated that there are approximately 800-1,000 diagnosed patients with TTR-CM worldwide, though the actual number of patients with TTR- CM is presently unknown." Uitgaande van 1.000 patiënten wereldwijd is de verwachting dat het in Nederland zal gaan om maximaal 10 patiënten. Echter gezien de verwachte MW en het ontbreken van andere alternatieven is de verwachting dat dit geneesmiddel bij een veel grotere groep ingezet gaat worden (schatting: kan oplopen tot tot 5.000 patiënten).

Expected cost per patient per year

Cost 90,000.00 - 110,000.00
References medicijnkosten.nl, GIP-databank
Additional remarks De kosten per capsule van 20 mg betreffen € 385,86 (medicijnkosten.nl). Vergoeding per gebruiker per jaar betreft zo'n €100.000,00 voor de nu al geregistreerde indicatie (GIP-databank). De verwachting is dat de kosten p.p.p.j. voor deze indicatie in dezelfde range komt te liggen.

Potential total cost per year

Total cost

250,500,000.00

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 No

Other information

There is currently no futher information available.