The divergence between the rapid pace of scientific progress in rare diseases and declining sentiment towards orphan medicines reflects a paradox that is at the heart of the European orphan drug debate. While the last twenty years have witnessed a... View More
The European Commission is currently reviewing the 2000 Regulation on orphan medicinal products (OMP). It is critical that any consideration of reform of the Regulation should be informed by a robust understanding of the relationship between incentives, investment, innovation and... View More
Gene and cell therapies represent some of the most advanced treatments being employed in biomedical research to improve the length and quality of life of patients with rare and complex diseases. Patients’ access to these therapies has been hampered by... View More