BACKGROUND: While new expensive medicines often offer substantial benefits to patients, they can carry inherent drawbacks such as uncertainty regarding efficacy translating into effectiveness, safety and rational use, as well as a substantial financial burden on society and/or patients. Rational use of medicines aims to maximize effectiveness whilst minimizing side effects, patient burden, and societal costs. In the Netherlands, initiatives aiming to improve the rational use of expensive medicines are being carried out with increasing frequency and in a programmatic manner. This review identified the strategies used, and includes a structured approach for their application during the medicine's use in clinical practice. MAIN BODY: Rational use initiatives, driven by clinicians and pharmacists, and funded by the Dutch Ministry of Health and the Dutch health insurers were evaluated for strategies that aim to improve the rational use of expensive medicines. In addition, a non-systematic narrative review was carried out through searches in Google, Google Scholar, Pubmed, the Artificial Intelligence (AI)- tools Global Campus and Evidence Hunt to identify additional strategies. Identified strategies were categorized by assessing whether they aimed to address the efficacy-effectiveness gap or to reduce the side effects and societal burden of expensive medicines. Thirteen strategies to improve rational use were identified. Two strategies were identified that aim to address the efficacy-effectiveness gap: optimize patient selection and generating evidence on clinical endpoints. 11 additional strategies that aim to reduce side effects or societal burden were identified: dose reduction, personalized dose optimization, interval lengthening, shortening of the treatment duration, biosimilar/generic drug use, non-medical drug switching, reduction of additional non-medication costs, reduction of drug wastage, switching the route of administration, boosting (improve drug exposure and/or reduce the dose by influencing pharmacokinetic parameters through co-interventions), and optimization of medication adherence. CONCLUSIONS: Rational use of expensive medications is essential as part of a drug's life cycle and can benefit patients as well as society. The framework and strategies described in this overview provide guidance for the future rational use of expensive medicines, both for those already in use and for those newly introduced.
Penninx et al. (Thu,) studied this question.