At RARE-NL, you can reach out with questions about gaining access to medicines. This applies to doctors and researchers, but also to institutions, companies, governments, and patient organizations. These questions may range in scope and relate to any part of the drug development chain. Examples include:

• What steps do I need to take to go from preclinical to clinical?
• Is testing the drug in a cell model sufficient, or do I need to conduct animal studies as well?
• Can I find funding for research with a patented product?
• How do I approach manufacturers effectively?
• What is my role as a healthcare provider in applying for reimbursement of a drug by a health insurer?

Sometimes, questions go beyond typical areas of expertise, such as:

• Is it wise to use an immediately available raw material in my research, or should I use a GMP-qualified one?
• A vitamin derivative is marketed as a “medical food” in the US — how can I get it reimbursed for my patient with a rare disease in the Netherlands?

Often, we’ve already answered similar questions, which can be found on the FAST forum or in our knowledge base. We also share knowledge through education, conferences, and events we organize ourselves. If we don’t have the answer, we refer you to a relevant expert.

Matchmaking

In drug repurposing—using an existing medicine for a new purpose—an interesting phenomenon occurs: many researchers don’t realize others are working on similar ideas in different disease areas. They could learn from one another or even collaborate.

While repurposing often starts out as disease-specific research targeting a known compound, advancing it to preclinical or clinical phases requires broader expertise. Sometimes, another party has already acquired this knowledge and is willing to share it—especially regarding regulations and funding. Matchmaking—connecting people who can benefit from each other’s experiences—is therefore a key part of our advisory role, and we even facilitate it within local university medical centers.

How can RARE-NL help you combine affordability and innovation in medicines for rare diseases or drug repurposing?