What should we consider when determining if an oncological therapy has clinical benefit? Find out with this recap of a 2021 European Society for Medical Oncology (ESMO) Congress session.
It’s not uncommon for oncologists to question how a treatment may impact patients, which is why the “Does My Patient Really Benefit from My Treatment? Physician, Patient, & Value Perspectives” session on day 6 of the European Society for Medical Oncology (ESMO) Congress featured the following three presentations exploring different perspectives on this topic.
Real-World Experience Using the ESMO-MCBS in Clinical Practice
Kicking off the session was medical oncologist Dr. Barbara Kiesewetter from Vienna, Austria. She and her team at the Medical University of Vienna have firsthand experience using ESMO’s Magnitude of Clinical Benefits Scale (ESMO-MCBS), which is a tool designed to highlight treatments that improve duration of survival and quality of life among cancer patients.
The first version of ESMO-MCBS was published in 2015, and to assess the clinical applicability and feasibility of the tool, a study was done at the Medical University of Vienna using the following three steps:
Assess therapies in regular use
Score them using the ESMO-MCBS
Assess results for the feasibility and plausibility of the scoring as well as the applicability of the tool
Dr. Kiesewetter found the tool to be reliable and reproducible in daily clinical practice, but it wasn’t without some limitations. Fortunately, the dynamic nature of the ESMO-MCBS tool allows for—and encourages—revisions based on feedback, and after two years, version 1.1 was published and addressed many of the limitations of version 1.
Dr. Kiesewetter’s team is currently using the latest version of ESMO-MCBS, and according to her, the score cards that provide ratings for EMA and FDA-approved drugs not only allow for real-time application of those drugs, but also more in-depth discussions with patients regarding treatment decisions.
Based on Dr. Kiesewetter’s experience, she views ESMO-MCBS as a dependable tool that enables oncologists to analyze if a treatment will result in clinical benefit, which in turn can help them improve patient survivorship and quality of life.
When Does Statistical Significance Become Clinically Relevant?
Up next was Mr. Mike Clarke from Belfast to talk about how researchers and oncologists can determine if a clinically relevant result is due to chance.
According to Mr. Clarke, randomized trials are done to determine what would happen if a treatment was not used. Not only do these trials help predict what would happen in similar circumstances in the future, but they can also help resolve important uncertainties, such as what treatment works best for whom and by how much.
However, the results of these experiments may be due to chance; this is especially true for trials where patients are randomly allocated to treatments. That’s why statistical significance is used to help determine if the result occurred by chance.
Statistical significance can be assessed by looking at the p-value, or the probability that the result occurred by chance, and the confidence interval. Once statistical significance is determined, then clinical relevance is assessed, meaning the likelihood of that result benefiting patients is examined.
This assurance that a result is both statistically significant and clinically relevant is key to oncology research, which is why Mr. Clarke provided the following questions to keep in mind:
- Is the finding clinically relevant?
- Is the finding statistically significant?
- Is the finding convincing?
Mr. Clarke then concluded the presentation by reminding attendees that it’s important to find the balance between statistical significance and clinical relevance and that chance effects should never be ignored.
Clinical Benefit from a Patient’s Perspective
Wrapping up the session was patient advocate Ms. Bettina Ryll from Uppsala, Sweden, who shared how her personal experience caring for her husband who was diagnosed with melanoma changed her perception of clinical benefit.
According to Ms. Ryll, tumor size is often quoted as one of the main factors that determine a therapy’s clinical benefit, but that completely ignores the anatomical location, which can have a huge impact on a patient’s quality of life. Her husband, for instance, couldn’t use his right hand because of his melanoma, which took a big toll on him.
Ms. Ryll then shared a recent survey that asked stage IV melanoma patients what mattered most to them, and the biggest response to come out of that survey was “family.” Family and having a normal life was what mattered most to them, which has a direct impact on the way side effects, such as diarrhea and fatigue, are talked about.
Based on her own personal experience and findings like the survey above, Ms. Ryll believes the oncology community needs to look at the research and assessment methods in place and be critical of them. By doing that, the community as a whole can change the thinking around clinical benefit and focus on what matters most: the patient.