Rilonacept Medicine 08 June 2021 Extended indication In paediactric and adult patients with Recurrent Pericarditis ≥12 years Therapeutic value No estimate possible yet Total cost € 6,645,000.00 Registration phase Clinical trials Product Active substance Rilonacept Domain Cardiovascular diseases Reason of inclusion New medicine (specialité) Main indication Other medication for cardiovascular diseases Extended indication In paediactric and adult patients with Recurrent Pericarditis ≥12 years Current proprietary name Already available biosimilars / generics Proprietary name Manufacturer Regeneron Portfolio holder Mechanism of action Unknown Route of administration Subcutaneous Therapeutical formulation Injection Budgetting framework Extramural (GVS) Centre of expertise Additional remarks Werkingsmechanisme: "Recombinant fusion protein that blocks IL-1α and IL-1β signaling" Registration Registration route Centralised (EMA) Type of trajectory Unknown Particularity Unknown ATMP No Submission date 2023 Expected Registration 2024 Orphan drug No Registration phase Clinical trials Reimbursement Medicine sluice Additional remarks Indieningsdatum op basis van primary completion datum fase 3 studie in mei 2020. Therapeutic value Current treatment options Anti-inflammatoire geneesmiddelen. Therapeutic value No estimate possible yet This assessment does not indicate any potential inclusion in the package. Substantiation In de RHAPSODY fase 3 studie placebo-gecontroleerd, had 7% in de rilonacept groep een pericarditis recurrence, vergeleken met 74% in de placebo groep. Mediane tijd tot recurrence: hazard ratio, 0.04; 95% CI, 0.01 to 0.18; P<0.001. Een interleukine remmer zal niet de eerste keuze zijn in het geval van een recurrent pericarditis. De verwachting is dat colchicine eerder in de behandelrichtlijn gegeven zal worden. Duration of treatment Frequency of administration 1 times a week Dosage per administration 320 mg References NCT03737110 Additional remarks Expected patient volume per year Patient volume < 30 Market share is generally not included unless otherwise stated. Maximum patient volume for sluice References http://echocardiografie.nl/wp-content/uploads/2019/06/Pericarditis-2019.pdf Additional remarks Diagnose Pericarditis wordt zo'n 28 keer per 100.000 personen per jaar gesteld. Het aantal recidief pericarditiden wordt geschat op circa 300 per jaar. Een klein percentage hiervan zal mogelijk met dit geneesmiddel behandeld kunnen gaan worden. Dit zal maximaal 10% zijn. Hierdoor komen er maximaal 30 patiënten per jaar in aanmerking. Expected cost per patient per year Cost € < 221,500.00 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 https://www.pharmaceuticalcommerce.com/view/the-worlds-most-expensive-drug-alexion-pharmaceuticals-soliris-at-409500-per-year Additional remarks De jaarlijkse kosten in de Verenigde Staten worden geschat op $250.000 maximaal per patiënt. In Nederland is de prijs nog niet bekend. Potential total cost per year Total cost € 6,645,000.00 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 Inborn genetic disorders, Urticaria References Adis Insight Additional remarks Other information Additional remarks