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Global Landscape of Benefit–Risk Considerations for Medicinal Products: Current State and Future Directions

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Abstract

In the last decade there has been a significant increase in the literature discussing the use of benefit–risk methods in medical product (including devices) development. Government agencies, medical product industry groups, academia, and collaborative consortia have extensively discussed the advantages of structured benefit–risk assessments. However, the abundance of information has not resulted in a consistent way to utilize these findings in medical product development. Guidelines and papers on methods, even though well structured, have not led to a firm consensus on a clear and consistent approach. This paper summarizes the global landscape of benefit–risk considerations for product- or program-level decisions from available literature and regulatory guidance, providing the perspectives of three stakeholder groups—regulators, collaborative groups and consortia, and patients. The paper identifies key themes, potential impact on benefit–risk assessments, and significant future trends.

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Acknowledgements

We are grateful to Dr Zuby Okemefuna, funded by CSL Behring, for providing writing and editorial support including writing the draft, creating tables and figures, journal styling, handling revisions and proof reading.

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Authors and Affiliations

  1. Global Clinical Safety and Pharmacovigilance, CSL Behring, King of Prussia, PA, USA

    Max Waschbusch

  2. Center for Drug Evaluation and Research, US Food and Drug Administration, Silver Spring, MD, USA

    Lisa Rodriguez

  3. Quantitative Safety and Epidemiology, Novartis, Basel, Switzerland

    Andreas Brueckner

  4. Center for Drug Evaluation and Research, US Food and Drug Administration, Silver Spring, MD, USA

    Kerry Jo Lee

  5. Center for Devices and Radiological Health, US Food and Drug Administration, Silver Spring, MD, USA

    Xuefeng Li

  6. Analytics and Data Sciences, Biostatistics, Biogen, Cambridge, MA, USA

    Oksana Mokliatchouk

  7. Quantitative Sciences and Reporting, CSL Behring, King of Prussia, PA, USA

    Lothar Tremmel

  8. Oncology Statistics, GlaxoSmithKline Plc, Upper Province, PA, USA

    Shuai S. Yuan

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WM and TL are employed by CSL Behring. BA, MO, and YS are employed by Novartis, Biogen and Kite Pharma, respectively. BA holds Novartis shares. They have no other conflicts to disclose. This publication reflects the views of the authors and should not be construed to represent their employers’ views or policies. RL, LKJ, and LX are employed by the US FDA. They have no other conflicts to disclose. This article reflects the views of the authors and should not be construed to reflect the FDA's views or policies.

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WM, RL, BA, LK, LX, MO, TL, and YS substantially contributed to all aspects of authorship, including conception and design, data acquisition, analysis, and interpretation; drafting and revising the work; final approval of the published version; and agreement to be accountable for all aspects of the work.

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Waschbusch, M., Rodriguez, L., Brueckner, A. et al. Global Landscape of Benefit–Risk Considerations for Medicinal Products: Current State and Future Directions. Pharm Med 36, 201–213 (2022). https://doi.org/10.1007/s40290-022-00435-x

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