Assessment and Benchmarking of Model Informed Approaches in Drug Development for Hemoglobinopathies: A Review of Scientific Advices From January 2000 to December 2024.

This publication represents an important milestone for the ERAMET project and highlights the growing role of model-informed drug development (MIDD) in advancing therapies for hemoglobinopathies, including conditions such as sickle cell disease and thalassemia.

The study provides a comprehensive and systematic review of scientific advice issued over a 25-year period, offering valuable insights into how modeling and simulation approaches have been applied in regulatory interactions. By benchmarking these approaches, the work identifies key trends, common challenges, and opportunities to further strengthen the use of quantitative methods in drug development.

Importantly, the findings underline the increasing recognition of model-informed strategies as powerful tools to support decision-making, optimize clinical trial design, and ultimately accelerate the development of effective treatments for patients with rare hematological disorders.

This achievement marks a significant step forward for Grace Shalom Govere and reflects the collaborative efforts within the ERAMET project to promote innovation in regulatory science and drug development.

We warmly congratulate Grace and all contributors involved in this work and look forward to building on these results in future research.

Read the full paper here.