Romosozumab Medicine 12 June 2018 Extended indication Osteoporosis in postmenopausal women and in men at increased risk of fracture. Therapeutic value Possible added value Total cost € 28,500,000.00 Registration phase Registration application pending Product Active substance Romosozumab Domain Metabolism and Endocrinology Reason of inclusion Main indication Other metabolism and Endocrinology Extended indication Osteoporosis in postmenopausal women and in men at increased risk of fracture. Current proprietary name Already available biosimilars / generics Proprietary name Evenity Manufacturer UCB Portfolio holder Mechanism of action Unknown Route of administration Subcutaneous Therapeutical formulation Injection Budgetting framework Extramural (GVS) Centre of expertise Additional remarks Monoclonal e antistof gericht tegen sclerostine, first-in-class. Samenwerking UCB en Amgen. FRAME Ext trial (reduced fracture risk). Registration Registration route Decentralised Type of trajectory Normal trajectory Particularity New medicine ATMP Unknown Submission date January 2018 Expected Registration February 2019 Orphan drug No Registration phase Registration application pending Reimbursement Medicine sluice Additional remarks Therapeutic value Current treatment options Calciferol, calciumcarbonaat, alendroninezuur, risedroninezuur, zoledroninezuur, denosumab Therapeutic value Possible added value This assessment does not indicate any potential inclusion in the package. Substantiation FRAME studie (Cosman et al): RCT, postmenopauzale vrouwen met osteoporose, romosozumab vs placebo 1e jaar, gevolgd door vergelijking met denosumab (in NL 2e lijnsbehandeling na bisfosfonaten): na 12 en 24 maanden incidentie van wervelfracturen verminderd (0.5% vs 1.8% en 0.6% vs 2.5%). Geen significant effect op niet-wervelfracturen ARCH trial: RCT 4093 postmenopauzale vrouwen met osteoporose romosozumab sc of alendronaat (70 mg) gedurende 12 maanden, gevolgd door openlabel alendronaat in beide groepen. Romosuzumab had meerwaarde tov alendronaat (wervelfracturen 50% en andere fracturen 19% verminderd tov alendronaat). Maar ernstig veiligheidsprobleem: cardiovasculaire complicaties bij 2.5% vs 1.9%. Discussie over oorzaak mogelijke nadelige effecten in NEJM en noodzakelijke aanvullende studies. NB: in FRAME studie 2 gevallen van kaakosteonecrose. Duration of treatment Average 12 month / months Frequency of administration 1 times a month Dosage per administration 210 mg References Cosman et al. N Engl J Med 2016; 375:1532-1543 Saag KG et al. N Engl J Med 2017;377:1417-27 Langdahl et al. Lancet. 2017 Sep 30;390(10102):1585-1594. Additional remarks Behandelschema: 120 mg 1 maal per maand gedurende 12 maanden, daarna 60 mg 1 maal per 6 maanden Expected patient volume per year Patient volume 1,500 Market share is generally not included unless otherwise stated. Maximum patient volume for sluice References GIPdatabank Additional remarks Denosumab zit nu op 20 miljoen met ruim 20000 gebruikers (voor alle indicaties) en Teriparatide op 5 miljoen met 1500 gebruikers. Als de veiligheidskwesties opgelost zijn, dan is de verwachting dat een vergelijkbaar volume als voor teriparatide zal gelden in Nederland. Mogelijk meer, als de richtlijn van de beroepsgroep adviseert het eerder in te gaan zetten. Expected cost per patient per year Cost € 18,000.00 - 20,000.00 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 $1700 per injectiepen in de VS. Potential total cost per year Total cost € 28,500,000.00 Total cost for sluice Additional remarks Off label use Off label use Unknown Indications off label use References Additional remarks Indication extension Indication extensions Unknown Indication extensions References Additional remarks Other information Additional remarks