Besides knowledge sharing, RARE-NL also provides support for concrete follow-up steps in the development process. A relatively simple example is applying for an Orphan Drug Designation from the European Medicines Agency (EMA). This status offers several advantages, such as exemption from registration fees, tax deductions for research and development costs, and a defined period of market exclusivity. A PhD candidate or a member of the relevant research group prepares the application together with someone from the RARE-NL network. In some cases, we also involve an external expert.

Unfortunately, we are not able to offer all services free of charge. Our contribution can be funded in various ways: sometimes through an invoice based on hours worked, or by co-authoring a (grant) application where, if successful, we are partially funded ourselves. In other cases, our input may take the form of an in-kind contribution.

For RARE-NL, it is essential to apply clear and consistent principles in order to achieve effective and fair collaboration. These principles are reflected in a public-private collaboration agreement that strikes a sound balance between societal value and financial sustainability. In short, our approach is built on four key areas:

1) Ownership & Risk Sharing – Project-specific entities ensure balanced ownership. Academic partners lead the research, while private partners manage product development.

2) Data & Evidence Generation – Data is accessible to all parties, in compliance with GDPR and FAIR principles.

3) Transparency & Licensing – Pricing is based on cost with a reasonable margin. Revenues are reinvested into drug development.

4) Patient Access – When benefit is proven, treatments remain accessible to patients.

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