The patientswe serve and their relatives are often members of a patient organisation. These organisations therefore have a very thorough knowledge of rare diseases and treatment strategies.

A rare disease affects a small percentage of the ­population, and is often serious, life-treathening or chronically debilitating. Many rare diseases appear early and are present throughout a person’s life. To date, the cause remains unknown for many rare ­diseases.

In Europe and North America, an estimated 60 million people are impacted by one of approximately 7,000 known rare diseases. Although more and more therapies are becoming available, the majority of rare diseases are without treatment. 

The history of orphan drugs developed to treat rare diseases is one of shared responsibility and ­community collaboration to achieve legislation and incentives to support the research and development of treatments. The main driver of this growth is successful legislation, combined with technological and scientific advancements, academic partnerships, the increasing availability of infra­structure, together with patient-led international and cross-border collaborations, which have helped to spur momentum worldwide.

Engagement with patient organisations

We recognise the importance of learning from patients and their families about the challenges they face, and the success and limitations of their current treatment options, in order to develop and deliver treatments that help meet their needs.

Collaboration is an integral part of our research, ­clinical programmes, patient access and pricing model. We support and partner with a wide range of patient organisations, both nationally and regionally, to reach the common goal of achieving the best patient outcomes. We do this by conducting research and raising awareness, and by working collaboratively with all stakeholders, including governments and healthcare systems. By working this way, we believe that we can not only bring treatments that will make a difference but that will reach the patients in a timely and sustain­able way. All interactions with patient organisations are made public on our website.

External links

The European Union-Orphan Drug Regulation

EURORDIS (Rare Diseases Europe)

NORD National Organization for Rare Disorders (USA)