RARE-NL takes a unique approach to effectively address rare diseases and their therapies: from case to system. This approach has proven itself in recent years by supporting individual initiatives on their path from scientific research to actual therapies for patients. At the same time, systemic bottlenecks are identified and addressed, directing researchers and companies to relevant expertise.

Research pathways, especially in rare diseases, often involve much more than just scientific knowledge. Besides preclinical research and clinical development, scale-up, GMP production, market authorisation and reimbursement models such as the Orphan Drug Access Protocol, for example, also play a crucial role. Many researchers and companies have questions about these. RARE-NL responds to this need by quickly involving experts who can help with advice, liaison or guidance for each question. Sometimes a short informal advice is enough, but more extensive support is also regularly provided.

RARE-NL’s approach is flexible and tailor-made: each case receives the care and attention it needs. This can range from advice on a specific aspect to joint pathways where therapies are fully developed. Protection of intellectual property is carefully safeguarded in the process.

What makes this approach special is that individual cases serve not only as ends in themselves, but also as learning experiences for the wider system. A case can be used anonymously to inform other researchers and companies or to discuss regulatory bottlenecks, such as the authorisation and reimbursement of advanced therapies (ATMPs).

RARE-NL always keeps an eye on both the specific case and possible improvements in the healthcare system, with the ultimate goal of making effective therapies available to patients quickly, safely and responsibly. This case-oriented approach is a powerful driver of systemic change in healthcare.