A new article was recently published: “Moving Toward a Question-Centric Approach for Regulatory Decision Making in the Context of Drug Assessment”. Fondazione Gianni Benzi cooperated in this work focused on the regulatory decision-making process aimed to put medicines on the market.

The most intuitive question to establish the efficacy and safety of medicinal products for market access is the benefit/risk balance. The benefit/risk assessment also includes questions related to dose selection, clinical and nonclinical pharmacology, and drug quality.

However, there is currently no systematic approach in the regulatory setting to assess and establish the acceptability of alternative methods and data sources. In most cases, the sponsors tend to prioritize traditional data types and methods, which are well accepted by regulators. This, in addition to the absence of rigor in the use and validation of new data types and methods, and the limited training of assessors in related fields can lead to increased regulatory scepticism toward new data types and methods.

This paper discusses the value of explicitly redefining and restructuring the regulatory scientific decision making around the scientific question to be addressed. Four use cases were described.

The ecosystem proposed is based on three pillars:

• a repository connecting questions, data, and methods
• the development and validation of high-quality standards for data and methods
• credibility assessment

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