Real-World Evidence Acceptability and Use in Breast Cancer Treatment Decision-Making in the United States: Call-to-Action from a Multidisciplinary Think Tank

Sean Khozin; Nancy A Dreyer; Dominic Galante; Raymond Liu; Peter Neumann; Nathan Nussbaum; Joyce O'Shaughnessy; Debra Patt; Mothaffar Rimawi; Hope Rugo; Sara M Tolaney; Marisa Weiss; Adam Brufsky

doi:10.1007/s12325-025-03201-y

Real-World Evidence Acceptability and Use in Breast Cancer Treatment Decision-Making in the United States: Call-to-Action from a Multidisciplinary Think Tank

Adv Ther. 2025 Jul;42(7):2973-2987. doi: 10.1007/s12325-025-03201-y. Epub 2025 May 12.

Authors

Sean Khozin^{1

2}, Nancy A Dreyer^{3

4}, Dominic Galante⁵, Raymond Liu⁶, Peter Neumann⁷, Nathan Nussbaum⁸, Joyce O'Shaughnessy^{9

10}, Debra Patt¹¹, Mothaffar Rimawi¹², Hope Rugo¹³, Sara M Tolaney¹⁴, Marisa Weiss¹⁵, Adam Brufsky¹⁶

Affiliations

¹ Massachusetts Institute of Technology, Cambridge, MA, USA. sean@phyusionbio.com.
² Phyusion LLC, 447 Broadway, 2 Floor #1104, New York, NY, 10013, USA. sean@phyusionbio.com.
³ University of North Carolina, Chapel Hill, NC, USA.
⁴ Dreyer Strategies LLC, Newton, MA, USA.
⁵ Precision AQ, Gladstone, NJ, USA.
⁶ San Francisco Medical Center, Kaiser Permanente, Northern California, San Francisco, CA, USA.
⁷ Center for the Evaluation of Value and Risk in Health, Institute for Clinical Research and Health Policy Studies, Tufts Medical Center, Boston, MA, USA.
⁸ Verily, Durham, NC, USA.
⁹ Baylor University Medical Center, Texas Oncology, US Oncology, Dallas, TX, USA.
¹⁰ Sarah Cannon Research Institute, Nashville, TN, USA.
¹¹ Texas Oncology, US Oncology, Dallas, TX, USA.
¹² Baylor College of Medicine, Houston, TX, USA.
¹³ University of California San Francisco Helen Diller Family Comprehensive Cancer Center, San Francisco, CA, USA.
¹⁴ Dana-Farber Cancer Institute, Harvard Medical School, Boston, MA, USA.
¹⁵ Breastcancer.org, Ardmore, PA, USA.
¹⁶ University of Pittsburgh Medical Center, Hillman Cancer Center, Pittsburgh, PA, USA.

Abstract

Complementing randomized controlled trials, real-world evidence (RWE) from observational analyses can extend clinical insights in oncology. While healthcare stakeholders have published rigorous RWE frameworks and resources, a multidisciplinary think tank was established to further advance acceptance and use of RWE in treatment decision-making, with the focus on breast cancer (while recognizing relevance in oncology more broadly). Members discussed perceptions of RWE from a clinical perspective, across domains of data, methodology, and mindset, and "calls-to-action" for stakeholders. Agreement was reached on a primary "call-to-action," to develop clinically-relevant, patient-informed, real-world endpoints, and secondary "calls-to-action": establish a multidisciplinary consensus forum; publish examples of unique RWE value; build upon existing frameworks and resources; and tailor an approach for exhibiting utility to guideline bodies.

Keywords: Breast cancer; Real-world data; Real-world evidence; Treatment decision-making.

MeSH terms

Breast Neoplasms* / therapy
Clinical Decision-Making* / methods
Decision Making
Evidence-Based Medicine
Female
Humans
United States