About: Orphan drugs

Europe at the crossroad of Rare Diseases innovation: lessons from the past and future outlooks

In shaping the future of rare diseases policies all stakeholders should cooperate to create an environment that actively supports research and clinical development of orphan medicinal products (OMPs). This entails building synergies all across the innovation ecosystem to address the...

The complexity of R&D for orphan medicines

There are multiple challenges inherent to drug development for rare diseases, all consequences of the small number of patients. For most of these diseases, the body of pre-existing knowledge is extremely limited with a lack of crucial information such as...

From pandemic to rare disease, medical innovation is the answer

Living through a global pandemic, we can take some comfort from the enormous collaborative research and development response that is taking place to find a way out from under the shadow of the COVID-19 crisis. Scientists around the world are...