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
Manufacturer Regeneron
Route of administration Subcutaneous
Therapeutical formulation Injection
Budgetting framework Extramural (GVS)
Additional comments Werkingsmechanisme: "Recombinant fusion protein that blocks IL-1α and IL-1β signaling"

Registration

Registration route Centralised (EMA)
ATMP No
Submission date 2022
Expected Registration 2023
Orphan drug No
Registration phase Clinical trials
Additional comments 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
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.
Frequency of administration 1 times a week
Dosage per administration 320 mg
References NCT03737110

Expected patient volume per year

Patient volume

< 30

Market share is generally not included unless otherwise stated.

References http://echocardiografie.nl/wp-content/uploads/2019/06/Pericarditis-2019.pdf
Additional comments 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
References https://www.pharmaceuticalcommerce.com/view/the-worlds-most-expensive-drug-alexion-pharmaceuticals-soliris-at-409500-per-year
Additional comments 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

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.

Off label use

There is currently nothing known about off label use.

Indication extension

Indication extension Yes
Indication extensions Inborn genetic disorders, Urticaria
References Adis Insight

Other information

There is currently no futher information available.