Patients with a rare disease often have to wait before new treatments become available. Last week, Carla Hollak, director of RARE-NL, discussed this topic on BNR Nieuwsradio.

For orphan drugs in particular, the evidence at the time of market entry is often still uncertain. This is because patient populations are small, disease mechanisms are complex, and long-term data only become available over time. A system that requires full certainty before access is possible therefore does not align well with the reality of rare diseases.

At RARE-NL, we therefore advocate for a more dynamic reimbursement system for medicines. A system in which promising treatments can become available under predefined conditions, with controlled use in clinical practice, reassessment based on emerging evidence, and pricing agreements that can adapt as uncertainty evolves.

In doing so, we can help prevent uncertainty from automatically leading to delay, while working towards a system that is both careful and accessible.

The episode (Dutch) can be found here:
https://www.bnr.nl/podcast/beter/10600111/hoe-krijgen-we-een-eerlijker-systeem-voor-nieuwe-medicijnen-in-nederland