‘Clinically meaningful’ outcomes in cancer care extend beyond survival
Key takeaways:
- A qualitative study evaluated interpretations of “clinically meaningful” among patients with metastatic breast cancer.
- Providers need to understand patients’ goals of care, particularly those unrelated to survival.
A qualitative study yielded important insights about how patients, caregivers and clinicians define what truly is clinically meaningful.
People living with metastatic breast cancer were not familiar with the concepts of clinical meaningfulness and clinically meaningful outcomes, according to the multiphase, mixed-method study.
Additionally, focus group participants underscored the importance of meaningful outcomes beyond survival, indicating a need to better understand individual patient interpretations and goals of care when discussing clinical meaningfulness.
Researchers at Brown University Health conducted the focus groups and evaluated three themes:
- interpretations of clinical meaningfulness/clinically meaningful outcomes;
- treatment recommendations, preferences and decisions; and
- clinical practice implications.
Investigators conducted 22 focus groups that included 50 people living with metastatic breast cancer, 24 caregivers and 41 providers (11 oncologists, 13 advanced care providers, 17 oncology nurses) between March and June 2023.
Results showed people living with metastatic breast cancer and caregivers were not familiar with the concepts of clinical meaningfulness and clinically meaningful outcomes. Health care providers knew these terms but did not use them when discussing metastatic breast cancer treatment with patients.
Participants across groups noted the importance of other “meaningful outcomes” beyond survival, including quality of life, alleviation of symptoms and improvement in functioning. However, the discussions revealed definitions of “clinically meaningful” are highly individual.
“This goes back to those very first lessons we learn in medical school, about first knowing who our patients are,” Stephanie L. Graff, MD, FACP, FASCO, director of breast oncology at Brown University Health and Legorreta Cancer Center at Brown University, told Healio. “Once we understand their values and who they are outside our clinic, we can use that context to discuss the implications of the disease and its treatment.”
Healio spoke with Graff about the study’s findings, their implications and her team’s next steps in research.
Healio: How does the definition of “clinical meaningfulness” relate to patient care?
Graff: ASCO developed a set of guidelines in which they define a clinically meaningful outcome as one that improves survival by 20%. That’s not always true on an individual level for any given patient, but it is the standard used by clinical trialists. ASCO’s definition of clinically meaningful outcomes ultimately contributed to the thinking of how we design clinical trials. The issue of whether a trial meets its statistical endpoint has a big impact on whether a phase 2 study moves on to phase 3, and whether results of a phase 3 study end up becoming part of everyday clinical practice. We often see trials that might demonstrate a big impact in terms of the difference between the control and the intervention arms, but if that impact didn’t cross the prespecified alpha allotment and meet a statistical P value, it doesn’t end up taking hold of prescribing habits in daily practice. Does that ultimately mean that that drug wouldn’t have been a clinically meaningful treatment for a certain group of patients? I would argue that it doesn’t.
Healio: What motivated you to conduct this study?
Graff: Our motivation was to understand the environment and the culture that clinicians, patients and caregivers are sharing when they sit down to make these decisions about what comes next. We wanted to understand how often these conversations happen and how they change over time, particularly in the metastatic breast cancer setting.
Healio: What did you find?
Graff: Our key finding from this initial qualitative work was that patients and their caregivers are unfamiliar with the definition of clinical meaningfulness and clinically meaningful outcomes as defined by the health care mega-system. Another key finding was that although health care providers — both nurses and physicians — are a bit more agile at articulating what a clinically meaningful outcome is, they don’t necessarily use the phrase in their day-to-day conversations with patients. However, both groups acknowledged and emphasized that outcomes can be clinically important beyond just survival. Issues such as quality of life, improvement in symptoms, and improvement in function were very important and very dynamic in the experience of treatment and disease.
Healio: What do you have planned next in this research?
Graff: Our next step is trying to move this from qualitative work to more quantitative work — a larger-scale analysis of how this is done and how it’s implemented. We can then start to develop tools to that foster these dialogues more effectively.
Healio: How can oncologists discuss clinically meaningful outcomes with patients in a way that promotes individualized care?
Graff: Any time a patient has a new diagnosis of metastatic breast cancer or the disease progresses, it is a good opportunity to establish and re-establish what road you’re on and where you are going. The clinician might say, “Your scans show evidence that your disease is growing, and this is a moment when we need to talk about changing therapy.”
However, before doing that, it always helps me to go back to the beginning and make sure the patient and I are aligned on what is most important to them. Is it most important for them to live as long as possible? Or are they focused on being able to climb the stairs in their home, or take that vacation to Florida, or babysit their grandchildren? Those are very different goals, and they drive decisions about what kind of care a patient wants to receive.
Reference:
For more information:
Stephanie L. Graff, MD, FACP, FASCO, can be reached at sgraff1@brownhealth.org.
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