Head of International Affairs Department - Italian Medicines Agency / Hospital Pharmacist specializing in Regulatory Affairs
Choosing predictive and clinically relevant endpoints is a key aspect of drug development in the regulatory space. By assessing orphan drug development for sickle cell disease (SCD) over the past two decades, we observed that failures in clinical development were mainly found in phase 3 due to lack of efficacy on vaso-occlusive crises as the primary study endpoint. This was likely related to variable definitions and heterogeneity of pain scoring and treatment. If you are interested in improving the debate on how fostering pharmaceutical R&D on SCD (and other rare diseases), you may wish to read our last work on this. https://lnkd.in/difgpBep #sicklecell #orphandrugs
