We are happy to answer your questions about medicines for rare diseases and the repurposing of (all) medicines. Our level of involvement can vary and is represented in a pyramid. At the base of the pyramid is our advisory role; at the top is the establishment of a joint project-based company (project-bv). We walk through the four tiers of this pyramid:

1. The helpdesk

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 a key part of our advisory role, and we even facilitate it within local university medical centers.

2. Non-binding advice

Sometimes, submitted cases are too complex for a simple answer.

In such cases, we use a structured format: the project advice. We begin with one or two intake meetings using a quick scan, which includes questions like:

• What exactly is the case?

• Are other parties already involved?

• Can a problem owner be identified?

• Is it too early, or is it time to start thinking about patient access?

Once we have mapped the case, we organize a meeting with relevant experts. This can involve topics such as regulatory affairs, ethics, production, or business development. As a national hub, RARE-NL can bring experts from across the country to the table.

Based on this discussion and preparatory work, we draft a concise advice outlining how the product could reach patients, which steps need to be taken, and how RARE-NL could be involved. A crucial aspect is the timeline—when is the right time to apply for orphan drug designation? Is a public-private partnership a suitable path, and when should it start? What must absolutely not be forgotten (e.g., intellectual property)? This leads us to the next levels of the pyramid.

3. Collaboration

The next tier is direct collaboration with RARE-NL. A relatively simple example is preparing an orphan drug designation application at the European Medicines Agency (EMA). This status offers benefits such as waived registration fees, tax relief for R&D costs, and a period of market exclusivity.

A PhD candidate or someone from the research group typically prepares the application in collaboration with a RARE-NL network member. Sometimes, we bring in an external expert.

Unfortunately, not all services can be offered for free. Our involvement can be funded in various ways—sometimes through direct invoicing for hours worked, sometimes by co-authoring a grant application and being partially funded upon success. In some cases, our contribution is made as an in-kind investment.

RARE-NL values clear and consistent principles for effective and fair collaboration. These are formalized in a public-private partnership agreement that balances social impact and financial sustainability, focusing on four key areas:

• ownership & risk sharing – project-bvs share ownership fairly. Academic partners lead the research; private partners handle product development.

• data & evidence – data is accessible to all parties under GDPR and FAIR principles.

• transparency & licensing – pricing is cost-based with fair profit. Revenues are reinvested into drug research.

• patient access – proven treatments remain accessible to patients.

4. Project-based company (project-bv)

When a collaboration becomes serious, we may establish a joint project-based company. This is grounded in the principle of steward ownership.

In these project-bvs, there are economic shareholders and stewards. Economic shareholders manage daily operations and receive returns on their investment. Stewards safeguard the structure of the company, deciding if and how it can be sold, at what price, and with whom partnerships are formed.

The RARE-NL foundation can act as a steward, ensuring the company adheres to the social principles outlined in section 3.

Socially driven investors are often eager to work with RARE-NL in this way. Together, we move towards making tangible medicines available and learning how to truly translate social values into practical outcomes.

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