Commentary on Hey, Hunter, Muller et al. A Never-ending Story: Evaluating Evidence in Medicinal Product Decision Making

Abstract

Decision making in medicinal product development and regulatory approval for marketing authorization are intended to be evidence based. While decision making based on evidence is documented to be a millennial old process, particularly for making legal and economic determinations, the nature and use of evidence continues to evolve, particularly when new technologies need to be factored in. The formal use of evidence for medicinal product evaluation has been in practice for less than 75 years and remains a dynamic process among all stakeholders. The nature of decision making for marketing authorization is particularly complex as it occurs on at least five levels: Causative, Generalizability, Clinical Significance, Risk to Benefit, and Value. Consequently, all stakeholders including product developers and regulatory authorities must communicate and coordinate to ensure functionality, as all are constantly learning to integrate new technologies, ever increasing knowledge of biology and disease, and improved understanding of the decision making process and its consequences. Structured interactions in the form of Scientific Advice with the European regulatory authorities were developed to facilitate knowledge sharing. The Scientific Advice process itself is dynamic and benefits from structured input on how it can better serve the needs and expectations of stakeholders. To that end, Hey, Hunter, Muller, et al provide suggestions in a manuscript following an initial consultative phase and then a peer review phase. The manuscript is an example of the type of engagement and good citizenship that can facilitate the efficient generation and use of evidence to support the complex decision making to which the evidence is applied.