Support for companies developing medicines for rare diseases is built into EU legislation through the Orphan Regulation, but these incentives are not always suitable for products that are being developed for very rare diseases or patient-specific treatments, experts from the European Medicines Agency and European Commission have heard.
During an interactive EMA webinar on orphan drug R&D and regulation in the EU, several officials from the EMA and the commission explained that they are well aware of the challenges faced by companies conducting N-of-1 trials, in which one patient is treated with a personalized medicinal product
