The potential application of an existing drug to a new indication is called Drug Repurposing. The potential of this relatively new route for innovative application is huge because the development process can be relatively shorter and cheaper than for traditional innovative drugs. Modern technology enables rapid screening for potential new applications but, at the same time, it appears that this route to the patient still has many bottlenecks. A healthy revenue model is often absent and limited regulatory options still too often hinder the route from initial discovery to consistent availability to patients. The limited successful examples of drug repurposing show both the huge untapped potential and the lack of a regular development route.

Rare diseases are the most common - that is, if someone has a disease, it is usually a rare disease. That fact is only getting stronger as our disease categories become more sophisticated. With the rise of personalised medicine, the number of orphan diseases will increase further. Thanks to incentives in this field, new orphan drugs have come onto the market in recent years, about 40% of them for rare cancers. However, the downside is that many of these drugs are expensive and even after patent expiry, the price hardly decreases due to insufficient competition (generic preparations or biosimilars). Conversely, for some rare diseases, simple but patient-critical treatments are available where sometimes there is limited incentive to keep these drugs available on a sustainable basis. The Netherlands is well placed to play a pioneering role in this field. Much research is being done in our country on disease mechanisms based on new models, (molecular) diagnostics but also on appropriate use and new ways to arrange early access for patients.