Supporting the development of rare disease R&D in Europe

摘要

In normal times, rare diseases don't always receive a high level of attention from the medical community. But, during the COVID-19 pandemic, it can seem that rare disease is even further down the agenda. "An incredible amount of attention has understandably been dedicated to COVID-19," says Nathalie Moll, Director General of the European Federation of Pharmaceutical Industries and Associations (EFPIA). "This has had an impact on those living with other health conditions, including rare diseases. Throughout the crisis, pharma companies have worked tirelessly to ensure the continuity of clinical trials in extremely challenging circumstances." But delays in access to orphan medicines are not a just a result of the COVID-19 crisis. "There has been a disparity of access for a number of years," says Moll. "It is not a COVID-19-related issue." Still, in the age of the coronavirus - and with around 30 million Europeans living with some form of rare condition - delivering new diagnostics and treatments for people living with rare conditions is more pressing than ever.