Extended indication Piqray is indicated in combination with fulvestrant for the treatment of postmenopausal women, and m
Therapeutic value Possible added value
Total cost 9,348,000.00
Registration phase Registered


Active substance Alpelisib
Domain Oncology and Hematology
Reason of inclusion New medicine (specialité)
Main indication Breast cancer
Extended indication Piqray is indicated in combination with fulvestrant for the treatment of postmenopausal women, and men, with hormone receptor (HR)‑positive, human epidermal growth factor receptor 2 (HER2)‑negative, locally advanced or metastatic breast cancer with a PIK3CA mutation after disease progression following endocrine therapy as monotherapy.
Proprietary name Piqray
Manufacturer Novartis
Mechanism of action Serine / threonine kinase inhibitor
Route of administration Oral
Therapeutical formulation Tablet
Budgetting framework Intermural (MSZ)
Additional comments α-specifieke class I phosphatidylinositol-3-kinase (PI3K) remmer.


Registration route Centralised (EMA)
Type of trajectory Normal trajectory
Submission date January 2019
Expected Registration July 2020
Orphan drug No
Registration phase Registered
Additional comments Positieve CHMP-opinie in juni 2020.

Therapeutic value

Current treatment options Diverse waaronder everolimus, palbociclib, letrozol, fulvestrant etc.
Therapeutic value Possible added value
Substantiation De SOLAR-1 studie laat een langere PFS zien ten opzichte van placebo. De overall response rate (ORR) in het PIK3CA-mutatie cohort was 26.6% in de alpelisib plus fulvestrant groep tegenover 12.8% in de placebo plus fulvestrant groep. Voor de totale studie populatie was de 'rate of grade ≥3 adverse events (AEs)' 76.1% in de alpelisib plus fulvestrant groep tegenover 35.5% in de placebo plus fulvestrant groep.
Duration of treatment Average 8.2 month / months
Frequency of administration 1 times a day
Dosage per administration 300 mg
References NCT02437318 (SOLAR-1); ESMO Abstract LBA3_PR
Additional comments 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.

References Fabrikant; ZiN Pakketadvies palbociclib, 11 april 2017; Mukohara. Breast Cancer (Dove Med Press). 2015 May 15;7:111-23.
Additional comments 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 patiëntvolume 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. Indien alle patiënten hier wel op getest zullen worden zal het maximale patiëntvolume 300 patiënten bedragen.

Expected cost per patient per year

Cost 29,520.00 - 32,800.00
References Fabrikant
Additional comments De fabrikant geeft aan dat de prijs tussen €29.520-€32.800 zal bedragen.

Potential total cost per year

Total cost


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

Off label use No

Indication extension

Indication extension Yes
Indication extensions Head and neck cancer; Ovarian cancer
References Adisinsight.
Additional comments Lopende fase 3 studies.

Other information

There is currently no futher information available.