Berkenschorsextract Medicine 08 December 2020 Extended indication Epidermolysis bullosa Therapeutic value Possible added value Total cost Registration phase Clinical trials Product Active substance Berkenschorsextract Domain Chronic immune diseases Reason of inclusion Indication extension IND Main indication Skin diseases Extended indication Epidermolysis bullosa Current proprietary name Already available biosimilars / generics Proprietary name Episalvan Manufacturer Amryt Portfolio holder Mechanism of action Unknown Route of administration Local Therapeutical formulation Gel Budgetting framework Extramural (GVS) Centre of expertise Additional remarks Registration Registration route Centralised (EMA) Type of trajectory Normal trajectory Particularity Unknown ATMP Unknown Submission date 2021 Expected Registration 2022 Orphan drug Yes Registration phase Clinical trials Reimbursement Medicine sluice Additional remarks Therapeutic value Current treatment options Palliatieve zorg Therapeutic value Possible added value This assessment does not indicate any potential inclusion in the package. Substantiation Er is momenteel geen behandeling voorhanden die de aandoening kan genezen. Duration of treatment Frequency of administration 1 times every 3 days Dosage per administration 10% References NCT03068780 Additional remarks De behandelingduur zal mogelijk duren tot wondgenezing. De toedeiningsfrequentie is 1 maal per 3-4 dagen. Expected patient volume per year Patient volume < 464 Market share is generally not included unless otherwise stated. Maximum patient volume for sluice References Baarman et al. J Eur Acad Dermatol Venereol. 2020 Oct 23. Additional remarks In Nederland zijn momenteel 464 patiënten bekend. Aangezien er momenteel geen goede behandelopties zijn zullen er veel van deze patiëntengroep in aanmerking kunnen komen. 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 Epidermolysis Bullosa is geen immunologische aandoening maar is toegevoegd aan het domein Chronische Immuunziekten vanwege technische redenen.