Rare diseases are more common than you might think That is to say: if someone has a disease, it is most likely a rare one. This reality is becoming even more apparent as our disease classifications become increasingly refined. With the rise of personalised medicine, the number of rare diseases (orphan diseases) is expected to grow further. Thanks to specific incentives in this field, a number of new orphan drugs have been brought to market in recent years — around 40% of which target rare forms of cancer.

However, many of these treatments are extremely costly, and even after their patents expire, prices tend to remain high due to a lack of sufficient competition (e.g. generic versions or biosimilars). At the same time, for some rare conditions, simple yet vital treatments exist, but there is little commercial incentive to keep these therapies sustainably available.

The Netherlands is well positioned to take a leading role in this field. A great deal of research is conducted in the country into disease mechanisms — often based on innovative models, advanced (molecular) diagnostics, appropriate use of therapies, and new approaches to ensuring early access for patients.

RARE-NL is committed to putting rare diseases higher on the agenda. As a national hub, RARE-NL promotes an integrated approach that connects research, regulation, development, and access. By bringing together stakeholders from science, healthcare, policy, and industry, RARE-NL supports concrete solutions that ensure sustainable access to treatments, better use of scientific knowledge, and structural improvements in patient access to care for rare diseases. More background information about medicines for rare diseases can be found here.