Alpelisib Medicine 13 December 2018 Extended indication Treatment of postmenopausal women, and men, with hormone receptor positive, HER2-negative, advanced breast cancer with PIK3CA mutation in combination with fulvestrant after disease progression following an endocrine-based regimen.ant Therapeutic value No judgement yet Total cost Registration phase Clinical trials Product Active substance Alpelisib Domain Oncology and Hematology Reason of inclusion New medicine (specialité) Main indication Breast cancer Extended indication Treatment of postmenopausal women, and men, with hormone receptor positive, HER2-negative, advanced breast cancer with PIK3CA mutation in combination with fulvestrant after disease progression following an endocrine-based regimen.ant Current proprietary name Already available biosimilars / generics Proprietary name Manufacturer Novartis Portfolio holder Mechanism of action Unknown Route of administration Oral Therapeutical formulation Tablet Budgetting framework Intermural (MSZ) Centre of expertise Additional remarks α-specifieke class I phosphatidylinositol-3-kinase (PI3K) remmer Registration Registration route Centralised (EMA) Type of trajectory Normal trajectory Particularity Unknown ATMP Unknown Submission date April 2019 Expected Registration April 2020 Orphan drug No Registration phase Clinical trials Reimbursement Medicine sluice Additional remarks Therapeutic value Current treatment options Diverse waaronder everolimus, palbociclib, letrozol, fulvestrant etc. Therapeutic value No judgement yet This assessment does not indicate any potential inclusion in the package. Substantiation De SOLAR-1 studie laat langere PFS zien ten opzichte van placebo. Duration of treatment Frequency of administration 1 times a day Dosage per administration 300 mg References NCT02437318 (SOLAR-1); ESMO Abstract LBA3_PR Additional remarks SOLAR-1: "Alpelisib (300 mg; oral; once daily) in combination with fulvestrant (500 mg; intramuscular injection on Day 1 and Day 15 of Cycle 1, and then Day 1 of each subsequent 28-day cycle)". Verwachte mediane behandelduur is minder dan 1 jaar Expected patient volume per year Patient volume < 300 Market share is generally not included unless otherwise stated. Maximum patient volume for sluice References Fabrikant; ZiN Pakketadvies palbociclib, 11 april 2017; Mukohara. Breast Cancer (Dove Med Press). 2015 May 15;7:111-23. Additional remarks Van de naar schatting 1.700 patiënten in 1e lijn, krijgt naar verwachting 70% (=1.190 patiënten) een behandeling in 2e lijn. Circa 30% hiervan heeft een PIK3CA mutatie en komt daarmee theoretisch in aanmerking voor alpelisib. Het verwachte patiëntenvolume ligt maximaal rond de 300. De vraag is wel of alle patiënten getest gaan worden voor deze mutatie en of ze in dat geval niet met een CDK4/6 remmer behandeld zullen worden. Om deze reden zal het uiteindelijke patiëntenaantal waarschijnlijk lager liggen dan 300. Expected cost per patient per year Cost References Additional remarks Potential total cost per year Total cost Total cost for sluice Additional remarks Off label use Off label use No Indications off label use References Additional remarks Indication extension Indication extensions No Indication extensions References Additional remarks Other information Additional remarks