Rational use of medicines

Even when a new medicine becomes available to patients, there are often improvements that can be made. Rational use means that the medicine is used in a way that ensures it is as effective as possible, causes the fewest side effects and discomfort for the patient, and keeps costs for society as low as possible. Ensuring rational use of medicines for rare diseases is particularly challenging. When a new drug is introduced to the market for a rare disease, there is usually less available evidence. Additionally, the severity of symptoms in rare diseases can vary widely, meaning that the most suitable treatment is not the same for every patient.

How can medicine use be more rational?

There are various ways to improve the rational use of medicines. For rare diseases, several indication committees have been established, where experts in a specific rare disease collaborate to determine the best possible treatment for patients. These committees consider the latest scientific research insights. Read more about indication committees in the report on indication committees – Zorginstituut Nederland.

Research plays a crucial role in ensuring rational medicine use. Various studies can be conducted, such as investigating whether a lower dosage is just as effective, exploring whether the intervals between medication administrations can be extended, and identifying specific patient subgroups that benefit most from treatment and those who do not.

Example of rational use

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.