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Moving Towards a Question-Centric Approach for Regulatory Decision-Making in the Context of Drug Assessment.

The most intuitive question for market access for medicinal products is the benefit/risk (B/R) balance. The B/R assessment can conceptually be divided into sub questions related to establishing efficacy and safety. There are additional layers to the B/R ratio for medical products, including questions related to dose selection, clinical and nonclinical pharmacology, and drug quality. Explicitly stating the actual questions and how they contribute to the overall B/R provides a structure that fosters better informed cross-domain discussions.

There is currently no systematic approach in the regulatory setting to assess and establish the acceptability of alternative methods and data sources. A data-knowledge backbone is needed to mitigate the uncertainty in efficacy and safety characterization. This white paper discusses the value of explicitly redefining and restructuring the regulatory scientific decision making around the scientific question to be addressed.

The ecosystem proposed is based on three pillars:

1. A repository connecting questions, data, and methods.
2. The development and validation of high-quality standards for data and methods.
3. Credibility assessment.

The ecosystem is applied to four use cases for illustration. The need for training and regulatory guidance is also discussed.

Recently, a consortium of academic, regulatory and industry partners submitted and were awarded a grant (HORIZON-HLTH-2023-IND-06-04) focused on “Maintaining an innovative, sustainable and globally competitive health industry” and specifically evaluating modelling and simulation to address regulatory needs in the development of orphan and paediatric medicines.

This paper highlights the needs and intentions around such an ecosystem and collaboration. The Aridhia DRE will be one of the central data sharing platforms supporting this initiative.

You can find the full text for the publication below.