A Survey to Assess the Current Status of Structured Benefit-Risk Assessment in the Global Drug and Medical Device Industry

Martin Gebel; Cheryl Renz; Lisa Rodriguez; Arianna Simonetti; Hong Yang; Brian Edwards; James Matthew Higginson; Nicola Charpentier; Michael Colopy

doi:10.1007/s43441-024-00650-5

A Survey to Assess the Current Status of Structured Benefit-Risk Assessment in the Global Drug and Medical Device Industry

Ther Innov Regul Sci. 2024 Apr 22. doi: 10.1007/s43441-024-00650-5. Online ahead of print.

Authors

Martin Gebel¹, Cheryl Renz², Lisa Rodriguez³, Arianna Simonetti⁴, Hong Yang⁵, Brian Edwards⁶, James Matthew Higginson⁷, Nicola Charpentier⁸, Michael Colopy⁹

Affiliations

¹ Statistics & Data Insights, Bayer AG, Aprather Weg 18a, 42113, Wuppertal, Germany. martin.gebel@bayer.com.
² Convene Pharma Consulting, LLC, Greater Chicago Area, USA.
³ Center for Drug Evaluation and Research, U.S. Food and Drug Administration, Silver Spring, MD, USA.
⁴ Center for Devices and Radiological Health, U. S. Food and Drug Administration, Silver Spring, MD, USA.
⁵ Center for Biologics Evaluation and Research, U.S. Food and Drug Administration, Silver Spring, MD, USA.
⁶ Husoteria Ltd. Ashtead, Surrey, UK.
⁷ Merck KGaA, Darmstadt, Germany.
⁸ BioNTech SE, Risk Management, Berlin, Germany.
⁹ UCB Biosciences, Research Triangle Park, NC, USA.

PMID: 38649524
DOI: 10.1007/s43441-024-00650-5

Abstract

Background: This industry survey was conducted to gain insight into the ways structured Benefit-Risk assessment (sBRA) of medical products is approached across drug or medical device developing companies, including frameworks and methods that are currently used and areas where future work is being planned.

Methods: A survey containing 28 questions covering five key areas of sBRA was set-up and shared with representatives from the participating companies. Each company was asked to complete a single survey response including inputs across the company's multidisciplinary key representatives involved in benefit-risk assessment.

Results: Of the 26 participating companies, 21 (81%) are conducting sBRA. Considering these 21 qualitative frameworks were used by almost every company (19, 90%), while only 12 (57%) have used a quantitative method. Many companies have sBRA training (17, 81%), document templates (16,76%), Standard Operating Procedures (SOPs)/checklists (13, 62%), and /or best practice manuals/examples (12,57%) available. Considering all 26 companies Software tools (15, 58%) and BR planning documents (11,42%) were identified as areas into which many companies intend to put effort.

Conclusions: The industry survey confirmed a wide usage of sBRA by many companies involved in research and development. Nevertheless, sBRA is evolving and several future opportunities like the implementation of visualization tools were identified by the representatives of the pharmaceutical companies. Finally, challenges like the cross-functional comprehension of the added value of sBRA are still seen.

Keywords: Decision making; Frameworks; Industry survey; Structured benefit-risk assessment.