Abatacept bij de behandeling van monogenetische en refractaire inflammatoire ziekte

Abstract

Abatacept is a potent anti-inflammatory drug. Its effectiveness and safety in the treatment of rheumatoid arthritis, psoriatic arthritis, and juvenile idiopathic arthritis has been well researched in randomized controlled trials. As healthcare providers for patients with rare inflammatory diseases, we believe that patients with CTLA4 deficiency and other rare immune disorders, where effectiveness is plausible, especially in cases of refractory disease, have a strong indication for treatment with abatacept. For patients with such extremely rare immunological or inflammatory conditions, conducting randomized controlled treatment trials is either not feasible or requires extraordinary effort. Consequently, reimbursement for these drugs often lags behind the pace of scientific advancements for many patients. We advocate for reimbursement of abatacept treatment for patients with monogenetic and refractory inflammatory diseases, to ensure that these patients receive the best possible treatment.