ABSTRACT
An effective benefit–risk (BR) assessment is core to both efficient product development and regulatory decision making, with the goal of being transparent, building trust, and enhancing communication across all stakeholders. Despite the diversity in tools and approaches used by stakeholders to assess and communicate a benefit–risk assessment, the use of a common BR approach can lead to better communication and optimal discussions. A common framework has been shown to improve the transparency, communication, and consistency of BR assessment, thereby making the underpinnings of implicit judgments and decisions more explicit. This is particularly important as value judgments and interpretations become more important in making not only regulatory decisions but also health technology assessment (HTA)/payer decisions. For a sponsor, a BR framework is a tool for planning the drug development process and a means for organizing, interpreting, and communicating the value proposition to various stakeholders, and a structured approach is also amenable to soliciting input from those stakeholders. The BR framework could also be used as a systematic means of building a database of regulatory decisions that can be accessed (beyond public assessment reports) by emerging agencies, HTA agencies, and patient groups. Because all parties seek clear guidance on the future use of BR assessment approaches, convergence is desired and achievable through activities such as the draft guidance developed by the International Conference on Harmonisation of Technical Requirements for Registration of Pharmaceuticals for Human Use (ICH) and shared experiences among stakeholders. A consistent BR framework provides one building block of quality decision making in pharmaceutical development and regulation upon which future practices and processes will be built that will facilitate and ensure that quality decisions are being made by all stakeholders.
