Areas of Interest for Investigator Sponsored Studies (ISSs)

The following areas of research are currently among those being considered to support novel, independent research that advances general knowledge for Sobi’s products. Research support is awarded on a highly competitive basis, and submission of research proposals in these areas of interest does not guarantee that support will be awarded. In order to enable scientific creativity, other research focus areas will be considered on a limited basis.

Anakinra (Kineret)

  • Clinical outcomes and predictors of response (e.g., biomarkers, patient characteristics) to anakinra therapy in rheumatoid arthritis
  • Clinical outcomes and predictors of response (e.g., biomarkers, patient characteristics) in macrophage activation syndrome (MAS) and secondary hemophagocytic lymphohistiocytosis (HLH)
  • Clinical outcomes and benefit-risk in idiopathic recurrent pericarditis (IRP)
  • Clinical outcomes in autoinflammatory diseases where no other validated therapies exist (e.g., familial Mediterranean fever, hyperimmunoglobulinemia D, TNF-receptor associated autoinflammatory syndrome, Schnitzler syndrome)
  • Proof of concept and efficacy studies for the role of anakinra to affect clinical outcomes in IL-1 driven diseases (Diseases must be well-characterized and have strong evidence for the role of IL-1 in their pathophysiology)

Nitisinone (Orfadin)

  • Clinical outcomes of Alkaptonuria (AKU)
  • Therapeutic monitoring approaches to hereditary tyrosinemia type 1 including dietary monitoring, nitisinone therapy monitoring, and newborn screening
  • Studies of adherence and compliance and association with clinical outcomes and quality of life
  • Non-drug management of HT1 patients (e.g., diet, co-morbidity management, education, etc.)