Even when a new medicine becomes available to patients, there are often opportunities for improvement. Appropriate use means that the medicine is used in a way that maximizes its effectiveness, minimizes side effects and patient burden, and keeps societal costs as low as possible.

Ensuring the appropriate use of medicines for rare diseases presents a challenge. When a new medicine for a rare disease is approved, there is generally less clinical evidence available. Additionally, the severity of symptoms in rare diseases can vary, meaning the most suitable treatment may not be the same for every patient.

How can medicine use be more rational?

1. Improved indication criteria (Which patient needs this medicine and at what time?)

In some cases, it is not entirely clear which patients truly benefit from a particular medicine. The use of these medicines can be optimized by improving indication criteria. Research can help determine which specific subgroup of patients within a disease benefits most from treatment and which subgroup does not.

For rare diseases, several “indication committees” have already been established. These committees consist of experts in a specific rare disease who collaborate to determine the best possible treatment for each patient, taking into account the latest scientific insights. Learn more about indication committees in the report: report on indication committees – Zorginstituut Nederland.

2. Reducing side Effects, patient burden, and costs

Once we are certain that a medicine is effective for a group of patients, we can explore ways to minimize side effects, patient burden, and costs. For example, research can determine whether a lower dose is just as effective or whether it is safe for patients to discontinue the medicine. This approach can help prevent unnecessary side effects, reduce hospital visits, and lower costs for society.

Fabry disease – developing start and stop criteria

Fabry disease presents significant variation in severity across different subgroups. Since it is inherited through the X chromosome, men typically experience more severe symptoms than women. Enzyme therapy with agalsidase alfa or agalsidase beta is effective but very costly and may cause side effects.

By establishing a database with data from 600 patients across three countries, subgroup-specific guidelines were developed to determine the optimal timing for initiating treatment. These guidelines considered factors such as gender and disease type (classical or non-classical). Additionally, recommendations were formulated to identify when enzyme therapy is no longer beneficial or may even be harmful. In such cases, supportive therapy was deemed the preferred course of action. The related publication can be accessed here.

How to fund research on rational medicine use

Several programs support research into the rational use of medicines and offer funding opportunities. In addition to local hospital initiatives, funding is available through specialized organizations such as KWF Kankerbestrijding (Dutch Cancer Society) and national programs, including the ZonMW Good Use of Medicines program, the Treatmeds Foundation, and the GIDS (Efficiency and Waste Reduction in Medicines) program within university medical centers (UMCs).

Questions?

If you have any questions about the rational use of medicines, please visit the FAST forum.