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)?

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