Hydroxycarbamide Medicine 08 December 2020 Extended indication Extension of indication to include treatment of severe chronic anemia (haemoglobin level < 6 g/dL or < 7 g/dL with poor clinical or functional tolerance) in adults, adolescents and children older than 2 years suffering from sickle cell syndrome. Therapeutic value Possibly no place in the treatment regimen Total cost Registration phase Registration application pending Product Active substance Hydroxycarbamide Domain Cardiovascular diseases Reason of inclusion Indication extension IND Main indication Other non-oncological hematological medications Extended indication Extension of indication to include treatment of severe chronic anemia (haemoglobin level < 6 g/dL or < 7 g/dL with poor clinical or functional tolerance) in adults, adolescents and children older than 2 years suffering from sickle cell syndrome. Current proprietary name Already available biosimilars / generics Proprietary name Siklos Manufacturer Addmedica Portfolio holder Mechanism of action Unknown Route of administration Oral Therapeutical formulation Tablet Budgetting framework Intermural (MSZ) Centre of expertise Additional remarks Registration Registration route Centralised (EMA) Type of trajectory Normal trajectory Particularity Unknown ATMP Unknown Submission date July 2020 Expected Registration August 2021 Orphan drug No Registration phase Registration application pending Reimbursement Medicine sluice Additional remarks Therapeutic value Current treatment options Hydrea Therapeutic value Possibly no place in the treatment regimen This assessment does not indicate any potential inclusion in the package. Substantiation Op dit moment wordt hydroxycarbamide (Hydrea) al off-label gebruikt. Er wordt geen gebruik van Siklos verwacht, zodra het geregistreerd is. Duration of treatment Frequency of administration 1 times a day Dosage per administration 20 mg/kg References NCT02516579 Additional remarks Expected patient volume per year Patient volume Market share is generally not included unless otherwise stated. Maximum patient volume for sluice References Additional remarks Er wordt geen gebruik verwacht. Expected cost per patient per year Cost € 9,337.00 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. References medicijnkosten Additional remarks Gemiddelde prijs per 30 stuks SIKLOS, TABLET 1000MG € 555,78. Wanneer we uitgaan van gemiddeld 1400 mg voor een patiënt van 70 kg. €778 per maand en €9.337 per jaar. 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