Dr. Rob ter Heine is a hospital pharmacist and clinical pharmacologist at Radboudumc, where is both active in the clinic and research. He heads the Radboud Applied Pharmacometrics group and is member of the Therapy Accelerator for Rare Diseases (part of RARE-NL). He focusses on bringing scientific questions from the bedside to the bench and back again, e.g. by implementation of model-informed precision dosing and academic model-informed drug development, thereby filling the many gaps that are left open by the commercial drug development system. He was awarded the His main research foci are lung cancer and rare/orphan diseases. Huizinga Penning in 2024, a price granted once every five years by the Dutch Association of Hospital Pharmacists in recognition of scientific contributions.
Model-informed precision dosing to improve treatment and cost-effectiveness of rare complement-mediated diseases
The complement system is an often-overlooked part of the immune system, responsible for various rare diseases. Two examples for these diseases are atypical hemolytic uremic syndrome (aHUS - a nephrological illness) and paroxysmal nocturnal hemoglobinuria (PNH - a hematological illness). aHUS is caused by genetic alterations in genes coding for complement regulation, PNH is caused by an acquired mutation in bone marrow cells, causing red blood cells triggering complement activation. aHUS can result thrombotic events in the kidney, as well as other organs and, when left untreated end-stage renal disease requiring transplantation. PNH results in life-threatening hemolysis and thrombosis when left untreated. Since approximately 10 years these diseases can be treated with therapeutic complement inhibitors. However, these drugs are expensive (>400.000 euro per person per year) and frequently not dosed optimally. Model-informed precision dosing can then help to simultaneously improve treatment and reduce costs. This presentation will focus on the development and implementation of model-informed precision dosing for complement inhibitors in collaboration with patients, healthcare professionals, government, and healthcare insurers to enable sustainable and personalized treatment.
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