JAK-remmers in de behandeling van zeldzame afweerstoornissen en autoinflammatoire aandoeningen

Abstract

JAK inhibitors are novel, powerful anti-inflammatory agents. For patients with relatively common inflammatory conditions such as rheumatoid arthritis and ulcerative colitis, the effectiveness and safety of these agents have been well studied in randomized controlled clinical trials. As healthcare providers for patients with rare immune disorders and autoinflammatory conditions, we see a strong indication for treatment with a JAK inhibitor in patients with a primary disruption of the JAK-STAT pathway and/or increased cytokine signaling via the JAK-STAT pathway. For patients with such ultra-rare immunological or inf lammatory conditions, conducting randomized controlled clinical trials is either not possible or requires extraordinary effort. As a result, reimbursement for these agents lags behind the pace of scientific and clinical advancements for many patients. Reimbursement for the treatment of JAK inhibitors for patients with an autoinflammatory condition or another very rare immunological condition where increased JAK/ STAT signaling plays a role in the pathophysiology is necessary to provide all patients with the best possible treatment.