How to monitor safety and efficacy of biologic treatment in rare, therapy-refractory immune-mediated inflammatory diseases?: Making the right clinical decisions for rare diseases with the right tools.
Anne Musters en Sander W. Tas
This article is an editorial in the scientific journal 'Rheumatology'. Evaluating the effects and safety of modern treatment agents which treat rare diseases can be complex, because it can be difficult to properly investigate them in a qualitative clinical trial due to the low number of patients with this disease. Therefore, it is important to adequately describe the effects of these treatments. This article suggests the five domains of the 'Rare IMID Disease Activity Score' (RIDAS).
Off-labelgebruik van biologicals voor zeldzame immunologische aandoeningen
Anne Musters, Amira Assaf, Dominique L.P. Baeten en Sander W. Tas
Patients with serious, immune-mediated inflammatory diseases (IMID's), like sarcoidosis, are increasingly treated with biologicals. Because research in these rare diseases is often very difficult, it is not known in all cases what the effects of the treatment are. In this article we elaborate on the RUBRIC-registry, in which we registrate the efficacy and the safety of these agents.