Selumetinib Medicine 12 June 2018 Extended indication Neurofibromatosis type 1 Therapeutic value No judgement Total cost Registration phase Clinical trials Product Active substance Selumetinib Domain Oncology and Hematology Reason of inclusion Main indication Neuroendocrine cancer Extended indication Neurofibromatosis type 1 Current proprietary name Already available biosimilars / generics Proprietary name Manufacturer AstraZeneca Portfolio holder Mechanism of action Unknown Route of administration Oral Therapeutical formulation Capsule Budgetting framework Intermural (MSZ) Centre of expertise Additional remarks Anti MAPK kinase (MEK). Registration Registration route Centralised (EMA) Type of trajectory Unknown Particularity New medicine ATMP Unknown Submission date 2018 Expected Registration 2019 Orphan drug No Registration phase Clinical trials Reimbursement Medicine sluice Additional remarks Therapeutic value Current treatment options Geen effectieve behandeling beschikbaar. Therapeutic value No judgement This assessment does not indicate any potential inclusion in the package. Substantiation Duration of treatment Average 30 month / months Frequency of administration 2 times a day Dosage per administration 25 mg/m2 References Dombi A. et al. N. Engl. J. Med. 2016 375:2550-60 Additional remarks Expected patient volume per year Patient volume < 6,000 Market share is generally not included unless otherwise stated. Maximum patient volume for sluice References NHG Additional remarks Er zijn in Nederland 4.000-6.000 patiënten met NF1. 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 Unknown Indications off label use References Additional remarks Indication extension Indication extensions Unknown Indication extensions References Additional remarks Other information Additional remarks