Rivipansel Sodium Medicine 12 June 2018 Extended indication Sickle cell disease; treatment of vaso-occlusive crisis. Therapeutic value No judgement Total cost Registration phase Clinical trials Product Active substance Rivipansel Sodium Domain Cardiovascular diseases Reason of inclusion Main indication Other non-oncological hematological medications Extended indication Sickle cell disease; treatment of vaso-occlusive crisis. Current proprietary name Already available biosimilars / generics Proprietary name Manufacturer Pfizer Portfolio holder Mechanism of action Unknown Route of administration Intravenous Therapeutical formulation Intravenous drip Budgetting framework Intermural (MSZ) Centre of expertise Additional remarks Small molecule glycomimetic inhibitor of E-, P- and L-selectins. Registration Registration route Centralised (EMA) Type of trajectory Unknown Particularity New medicine ATMP Unknown Submission date 2019 Expected Registration 2020 Orphan drug Yes Registration phase Clinical trials Reimbursement Medicine sluice Additional remarks Therapeutic value Current treatment options hydroxycarbamide Therapeutic value No judgement This assessment does not indicate any potential inclusion in the package. Substantiation Duration of treatment Frequency of administration Dosage per administration References Additional remarks Expected patient volume per year Patient volume < 450 Market share is generally not included unless otherwise stated. Maximum patient volume for sluice References http://www.hematologienederland.nl, https://www.sps.nhs.uk Additional remarks In Nederland leven naar schatting 1000 patiënten met SCZ(bron:hematologienederland). 90% van alle SCZ patiënten komt mogelijk in aanmerking voor deze behandeling (bron SPS). Gezien er nog een middel ontwikkeld wordt voor dezelfde indicatie is de inschatting dat maximaal 450 patiënten voor deze behandeling in aanmerking komen. Expected cost per patient per year Cost References GIP-databank Additional remarks Vergoeding per gebruiker in 2015 betrof zo'n 1750 euro voor hydroxycarbamide (GIP-databank). Potential total cost per year Total cost 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