Cannabidiol Medicine 10 December 2019 Extended indication Epidyolex is indicated for use as adjunctive therapy of seizures associated with tuberous sclerosis complex (TSC) for patients 2 years of age and older. Therapeutic value No estimate possible yet Total cost € 27,405,000.00 Registration phase Registered Product Active substance Cannabidiol Domain Neurological disorders Reason of inclusion Indication extension IND Main indication Epilepsy Extended indication Epidyolex is indicated for use as adjunctive therapy of seizures associated with tuberous sclerosis complex (TSC) for patients 2 years of age and older. Current proprietary name Already available biosimilars / generics Proprietary name Epidyolex Manufacturer GW Pharmaceuticals Portfolio holder Mechanism of action Cannabinoid Route of administration Oral Therapeutical formulation Solution Budgetting framework Extramural (GVS) Centre of expertise A small-molecule cannabinoid compound Additional remarks Registration Registration route Centralised (EMA) Type of trajectory Normal trajectory Particularity Unknown ATMP Unknown Submission date April 2020 Expected Registration April 2021 Orphan drug Yes Registration phase Registered Reimbursement Medicine sluice Additional remarks Positieve CHMP-opinie in februari 2021. Therapeutic value Current treatment options Anti-epileptica Therapeutic value No estimate possible yet This assessment does not indicate any potential inclusion in the package. Substantiation De therapeutische waarde lijkt op basis van de fase 3 studie aanzienlijk, echter is de invloed van de geneesmiddelinteractie mogelijk zorgelijk. Cannabidiol remt onder andere CYP3A4 en CYP2C9, wat respectievelijk clobazam en valproaat metaboliseert. Deze middelen zijn in de fase 3 trial door 45% en 27% van de patiënten gebruikt. Duration of treatment Frequency of administration 2 times a day Dosage per administration 25-50 mg/kg/dag References NCT02544763 Additional remarks Expected patient volume per year Patient volume 559 - 1,331 Market share is generally not included unless otherwise stated. Maximum patient volume for sluice References Brochure Tubereuze Sclerose Complex, NHG 2014 Chu-Shore, C., Major, P., Camposano, S., Muzykewicz, D., & Thiele, E. (2009). The natural history of epilepsy in tuberous sclerosis complex. Epilepsia, 51(7), 1236-1241. Additional remarks Naar schatting zijn er in Nederland ongeveer 1.500-2.500 patiënten met TSC. Ongeveer 70%-80% hiervan vertoont epileptische aanvallen (1.050-2.000). Alleen patiënten met refractaire epilepsie zullen in aanmerking komen voor deze behandeling. "85.2% had a "history of seizure'' en ''Of epilepsy patients, 62,5% developed refractory epilepsy'' (Chu-Shore et al, 2009). Dit komt neer op 85,2% van 1.050-2.500 is 895-2.130, waarvan 62,5% is een totaal van 559-1.331 patiënten. Expected cost per patient per year Cost € 29,000.00 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 FiercePharma Additional remarks In de Verenigde Staten is de lijstprijs gesteld op $32.500 (is omgerekend ongeveer €29.000). Potential total cost per year Total cost € 27,405,000.00 Total cost for sluice Additional remarks Off label use Off label use Unknown Indications off label use References Additional remarks Indication extension Indication extensions Yes Indication extensions Rett syndroom References Website GW pharmaceuticals Additional remarks Other information Additional remarks