At RARE-NL, we believe that collaboration is essential to accelerate the development of therapies for rare diseases and advance drug repurposing efforts. In a field full of complex challenges, innovative and sustainable solutions are needed. By bringing together academic institutions, patient organizations, and pharmaceutical and biotech companies—large and small—we create a value chain that spans the entire trajectory: from fundamental research to the eventual approval of medicines and patient access.

RARE-NL offers a platform where expertise on socially responsible access to these medicines converges. In the early stages of the development process, we can provide advice on what researchers need to consider to ensure that a drug is ultimately affordable and accessible to patients. As development moves towards the clinical phase, RARE-NL can assist in establishing public-private partnerships (PPPs) under socially responsible conditions. For medicines brought to market by industry, RARE-NL provides clinicians, pharmacists, and researchers with expertise on innovative reimbursement pathways.

In every case, we work to bring a medicine closer to the patient, always in a socially responsible manner. At the same time, we explore how these cases can offer insights to structurally improve the healthcare system.

Support services by RARE-NL

Sharing knowledge
RARE-NL facilitates knowledge sharing through various channels such as educational platforms, our website, events, presentations, and the FAST Forum. Questions can be directed to team members or submitted via the contact form.

Advice on cases
Where needed, we provide non-binding advice on specific cases, potentially with the involvement of external expertise. This may include development trajectories for medicines for rare diseases, drug repurposing, or reimbursement pathways.

Sustainable partnerships
We support the establishment of strategic and sustainable collaborations aimed at delivering innovative solutions and products. For instance, RARE-NL offers guidance on applying for support from organizations such as the EMA (European Medicines Agency) and CBG (Medicines Evaluation Board).

Increasing availability
By formalizing collaborations we strengthen collective impact and engagement. Think of initiatives towards a product-BV, whether or not financially supported by RARE-NL. For certain cases, this is the best way to ultimately ensure patient access at a socially acceptable price. RARE-NL can be involved in such BVs to safeguard societal interests.

Together, we are building a future where rare diseases no longer go untreated. Get in touch with RARE-NL and discover the value of this collaboration.