Library Header Image
LSE Research Online LSE Library Services

Advancing structured decision-making in drug regulation at the FDA and EMA

Angelis, Aris ORCID: 0000-0002-0261-4634 and Phillips, Lawrence D. (2021) Advancing structured decision-making in drug regulation at the FDA and EMA. British Journal of Clinical Pharmacology, 87 (2). 395 - 405. ISSN 0306-5251

[img] Text (Advancing structured decision-making in drug regulation at the FDA and EMA) - Published Version
Available under License Creative Commons Attribution Non-commercial No Derivatives.

Download (1MB)

Identification Number: 10.1111/bcp.14425


The recent benefit–risk framework (BRF) developed by the Food and Drug Administration (FDA) is intended to improve the clarity and consistency in communicating the reasoning behind the FDA's decisions, acting as an important advancement in US drug regulation. In the PDUFA VI implementation plan, the FDA states that it will continue to explore more structured or quantitative decision analysis approaches; however, it restricts their use within the current BRF that is purely qualitative. By contrast, European regulators and researchers have been long exploring the use of quantitative decision analysis approaches for evaluating drug benefit–risk balance. In this paper, we show how quantitative modelling, backed by decision theory, could complement and extend the FDA's BRF to better support the appraisal of evidence and improve decision outcomes. After providing relevant scientific definitions for benefit–risk assessment and describing the FDA and European Medicines Agency (EMA) frameworks, we explain the components of and differences between qualitative and quantitative approaches. We present lessons learned from the EMA experience with the use of quantitative modelling and we provide evidence of its benefits, illustrated by a real case study that helped to resolve differences of judgements among EMA regulators.

Item Type: Article
Official URL:
Additional Information: © 2020 The Authors
Divisions: LSE Health
Health Policy
Subjects: R Medicine > RS Pharmacy and materia medica
Date Deposited: 12 Jun 2020 12:51
Last Modified: 16 May 2024 13:43

Actions (login required)

View Item View Item


Downloads per month over past year

View more statistics