Off-labelgebruik van biologicals voor zeldzame immunologische aandoeningen

Anne Musters, Amira Assaf, Dominique L.P. Baeten en Sander W. Tas

Off-label use of biologics for rare immunological disorders

Patients with severe therapy-refractory immune-mediated inflammatory diseases (IMIDs) are increasingly treated with biologics. Since official practice guidelines for these rare diseases are lacking, these drugs are often prescribed off-label. However, treating physicians encounter difficulties and risks when prescribing drugs off-label. Access to these biologics for off-label indications is currently not well organized, which may result in suboptimal care. This also blocks innovation in patient care. In this article we introduce the recently initiated RUBRIC (Rational Use of Biologics in Refractory Immune-mediated inflammatory diseases Consortium) register. This national register documents the efficacy and safety of off-label biologic treatment in combination with the development of treatment protocols and practice guidelines. We believe that the results from this register will ultimately lead to a more evidence-based use of biologics in patients with rare IMIDs, which may also result in better access to these drugs. Ultimately, both the individual patient and the group as a whole will benefit.

Conflict of interest and financial support: the RUBRIC register is financially supported by unrestricted educational grants from AbbVie, Pfizer, Roche and MSD.