Skip to main page content
U.S. flag

An official website of the United States government

Dot gov

The .gov means it’s official.
Federal government websites often end in .gov or .mil. Before sharing sensitive information, make sure you’re on a federal government site.

Https

The site is secure.
The https:// ensures that you are connecting to the official website and that any information you provide is encrypted and transmitted securely.

Access keys NCBI Homepage MyNCBI Homepage Main Content Main Navigation
Review
. 2019 Aug 26:10:2042098619871180.
doi: 10.1177/2042098619871180. eCollection 2019.

Benefit-risk evaluation: the past, present and future

Juhaeri Juhaeri  1
Affiliations
Review

Benefit-risk evaluation: the past, present and future

Juhaeri Juhaeri. Ther Adv Drug Saf. .

Abstract

In the last two decades there has been a shift in the approach to evaluating the benefit-risk (BR) profiles of medicinal products from an unstructured, subjective, and inconsistent, to a more structured and objective, process. This article describes that shift from a historical perspective; the past, the present, and the future, and highlights key events that played critical roles in changing the field.

Keywords: benefit–risk; methods; patient; preference; quantitative; structured; weight.

PubMed Disclaimer

Conflict of interest statement

Conflict of interest statement: Juhaeri Juhaeri is an employee of Sanofi, a biopharmaceutical company that manufactures various drugs and biologics. While he did not receive a specific grant for this manuscript, he received salary and restricted shares from Sanofi. In addition, he owns an investment account which, at certain points in time, may include investment in biopharmaceutical business.

Figures

Figure 1.
Figure 1.
FDA benefit–risk framework. Adapted from Structured approach to benefit–risk assessment in drug regulatory decision making: draft PDUFA V implementation plan – February 2013 fiscal years 2013–2017. FDA, US Food and Drug Administration, PDUFA V, fifth authorization of the Prescription Drug-User-Fee Act.
See this image and copyright information in PMC

References

    1. Cocchetto DM, Nardi RV. Benefit-risk assessment of investigational drugs: current methodology, limitations, and alternative approaches. Pharmacotherapy 1986; 6: 286–303. - PubMed
    1. Aaron CS, Harbach PR, Mattano SSet al. Risk and benefit evaluation in development of pharmaceutical products. Environ Health Perspect 1993; 101(Suppl. 3): 291–295. - PMC - PubMed
    1. Rawlins M. Trading risk for benefit. Risk and consent to risk in medicine. London: Parthenon Publishing, 1989, pp.193–202.
    1. Edwards R, Wiholm BE, Martinez C. Concepts in risk-benefit assessment. A simple merit analysis of a medicine? Drug Saf 1996; 15: 1–7. - PubMed
    1. CIOMS. Benefit-risk balance for marketed drugs: evaluating safety signals. Geneva: CIOMS Working Group IV, Council for International Organizations of Medical Sciences (CIOMS), 1998, pp. 21–22.

LinkOut - more resources