Tafamidis Medicine 13 December 2018 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 IND Main indication Other medication for cardiovascular diseases Extended indication Transthyretin cardiomyopathy Current proprietary name Already available biosimilars / generics Proprietary name Vyndaqel Manufacturer Pfizer Portfolio holder Mechanism of action Unknown Route of administration Oral Therapeutical formulation Capsule, soft Budgetting framework Extramural (GVS) Centre of expertise Additional remarks Tafamidis is a kinetic stabilizer of TTR that inhibits misfolding and amyloid formation. Registration Registration route Centralised (EMA) Type of trajectory Unknown Particularity Unknown ATMP Unknown Submission date Expected Registration November 2019 Orphan drug Yes Registration phase Clinical trials Reimbursement Medicine sluice 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 This assessment does not indicate any potential inclusion in the package. Substantiation effect op sterfte en events Duration of treatment Frequency of administration 1 times a day Dosage per administration 20 mg / 80 mg References Additional remarks Expected patient volume per year Patient volume 10 - 5,000 Market share is generally not included unless otherwise stated. Maximum patient volume for sluice 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 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. 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 Total cost for sluice Additional remarks Off label use Off label use Unknown Indications off label use References Additional remarks Indication extension Indication extensions No Indication extensions References Additional remarks Other information Additional remarks