Hello. My name is Leonard Saltz. I'm a medical oncologist at Memorial Sloan Kettering Cancer Center in New York. I am chair of the GI oncology group as well as chair of the hospital's pharmacy committee.
Often we deal with this delicate problem: How do we maintain a hopeful posture while avoiding overhyping the results of our clinical trials and the progress that we've made? We need to strike a balance. We want to be supportive for our patients and hopeful where the science justifies that, but we have to be careful not to overpromise and be set up to underdeliver. We need to be thinking about communicating accurately what our clinical trial data say.
One of the places where this gets most confused is with the concept of a significant result. We use the term "significant" indiscriminately, without defining whether we're talking about statistical significance or clinical significance. When we say that a trial has a significant survival benefit, typically that means there is a statistically significant survival benefit. It may be a very limited number of days, but it may be real. The statistical significance tells us that difference is due to the drug and not just a chance finding.
Then we get into the more important question of clinical significance. What is a clinically significant outcome? We have many drugs on the market that are providing survival benefits of well less than 2 months—less than 60 days. I would argue that even if it's a statistically significant benefit, maybe it's not a clinically significant benefit within the context of the side effects of the drugs, inclusive of the financial toxicity inherent in the cost of these very expensive drugs.
When we talk to the patients and say that there's a significant survival benefit, we need to understand that they're going to hear that we just said there's a substantial survival benefit. A substantial survival benefit is going to mean different things to different people. We may need to quantify that. I don't think most people would argue that less than 2 months of survival benefit is not substantial relative to what we want to be able to offer our patients and relative to what our patients are expecting from us. I believe that a careful understanding of what a study is saying will help us communicate the information more accurately.
The other term that is widely misunderstood is "progression-free survival." That is not overall survival. This is a [concept] that I believe should be referred to as the progression-free interval. It's the amount of time from when we start a treatment until the treatment is no longer benefiting the patient, either because of progression of disease or because the patient died. I think we could agree that if the patient dies, the drug isn't working anymore. We don't need the term "survival" in the metric in order to understand what we're talking about.
"Progression-free interval" gives us a more accurate understanding of what we're talking about. "Progression-free survival" implies that people are living longer, and we don't know that. We can debate whether in some settings the progression-free survival may be an accurate surrogate for improved overall survival. It is not the same thing, and we need to be careful about understanding this in our thinking and our communicating to our patients. We can expect that if we tell a patient, "This improves the progression-free survival," that the patient is going to assume that this increases his or her chance of living longer, and that would be inaccurate.
Dealing with the hype has to do with putting accurate information on the table for discussion, getting rid of the taboos of conversation, and increasing transparency as to the strengths of a drug, its weaknesses, its toxicities and side effects, and its cost. What portion of that is going to be a financial burden on the patient?
All of this needs to be considered within the context of the alternatives. Are there less-toxic drugs that might be considered, perhaps with slightly less benefit, but with substantially better quality of life? Are there nondrug options? Are there supportive-care options? When in the course of treatment is it appropriate to say, "At this point we don't believe that a drug is going to help you"? How can we use the clinical trial data and put it in proper context? If a trial is done in a patient with an excellent performance status and good end-organ function, how do we help patients understand how that drug might not be able to contribute if they have a poor performance status and multiple organ dysfunctions or multiple medical comorbidities?
It's not going to be simple. There's no cookbook that's going to explain how to do this. This is part of the art of good oncology. I believe that being open, direct, and fair with our patients, and sharing the information with them as we understand it—making sure that we understand it accurately—is going to be the best way to approach it. Thank you.
Medscape Oncology © 2017 WebMD, LLC
Any views expressed above are the author's own and do not necessarily reflect the views of WebMD or Medscape.
Cite this: 'Dealing With the Hype': Communicating and the Art of Oncology - Medscape - Nov 02, 2017.