The future of Benefit-Risk assessments of medicinal products

Abstract

Introduction: Defining whether the benefit of a medicinal product outweighs its risk is a real challenge and needs to be assessed at several stages of the product life-cycle. Because the assessment is an important basis for decision-making for a broad group of stakeholders, it needs to be transparent and consistent. Furthermore, it is of interest to all stakeholders that the B-R assessment at the time of approval is predictive for the B-R profile of a marketed product. To increase predictability, the efficacy-effectiveness gap should be minimized.

Methods: Based on a literature study and discussions with experts, main challenges with the current way in which B-R assessments of medicinal products are performed were identified. Furthermore, possible ways to address these main challenges were analyzed. The latter is also done by a literature study and by talking to experts.

Results: Two main challenges with the current way of performing B-R assessments is the lack of transparency and consistency and the fact that the assessment at the time of approval is often not predictive for the B-R profile in a later stage. A more transparent and consistent way of performing B-R assessments can be achieved by using a descriptive/quantitative frameworks. Minimizing the efficacy-effectiveness gap can make the assessment of B-R at the time of approval more predictive for the B-R profile in a later stage.

Conclusion: Current challenges with the way in which B-R assessments are performed will lead to significant changes in the near future. MAHs should adapt to this changing environment, by using developed frameworks for the assessment of B-R or by addressing potential ways to minimize the efficacy-effectiveness gap. Addressing the problem of poor-adherence and making use of biomarkers will help MAHs in getting their products on the market and maintaining this market approval by ensuring a safe and effective use.