COMMENTARY

A Dying Patient's Request: Return My Trial Results

Mark A. Lewis

Disclosures

December 21, 2021

This transcript has been edited for clarity.

Hello. This is Mark Lewis for Medscape. I've been thinking about clinical trials and our responsibility to return results to participants. I come at this problem using both my head and my heart.

To step back, I know that oncologists have a little bit of a public image problem and that we might be viewed as mad scientists — as people in white coats who are experimenting on human subjects as if they are guinea pigs. I think that's an unfair portrayal, but I think that stereotype exists.

We all know that the engine of progress ­­— short of sheer luck — is methodical research, and that relies on ongoing human research beyond, say, murine studies, in order to advance our field. I think it's absolutely crucial that we recognize the importance of trust. People, specifically patients, have to trust us to enroll on these protocols.

When I sit at a conference or I read a journal and I see a Kaplan-Meier survival curve, I understand objectively what I'm looking at. I can process it cognitively, but from an emotional perspective, I realize that each declivity in that curve is not survival at all. It's patients who were lost, either to follow-up and censored or in the more colloquial sense — they died. I think that has to have an emotional impact, even while we strive to be objective scientists.

What I'm leading up to is that I recently had the opportunity to return the results of a trial posthumously. Several years ago, I enrolled a very fit young man, certainly a performance status of zero, in robust health, except for — and this may sound disingenuous — his stage IV cancer. We went over all his treatment options, and at the time, a trial of immunotherapy for his particular cancer type appeared that looked promising. He agreed to enroll, realizing that it may not be of individual benefit for him — and indeed it was not. His disease progressed with frightening rapidity, almost inexorably, and he died.

Before he died, he made me make a promise, and what he asked was very touching. He said, "Dr Lewis, once this study matures [in as many words], will you let my wife know how it turned out?"

We all know in oncology that not every trial is a positive trial. Our hypotheses of biological plausibility have to be sacrificed on the altar of data, and there are many negative studies. This one was not. This was positive. This showed a statistically and, I think, clinically significant therapeutic benefit for some of the enrollees — just clearly not this unfortunate young man.

I was of two minds in calling his widow, if I'm honest. On the one hand, I had a promise to fulfill. On the other hand, I didn't want to seem to her as if I was gloating that other patients had done better than her late husband. She could not have been more gracious. She said, "You know, he would have wanted to know that even one person was being helped by the research in which he participated, even if it didn't help him."

I just thought, that is such a selfless thing to say, so gracious in the face of grief. To step back, I know I'm citing an emotional — you might even say extreme — example, but I think we have our part of the social contract that we need to fulfill, and that is the return of results. What I see happen all too often is patients enroll in studies, and even the very participants in trials may not then be given the outcome, especially if that study is negative.

We all know that there's a publication trend toward positive studies getting more attention. I understand that, but I also think in the modern era — especially with our digital means of publication and dissemination — it's really unfortunate and, frankly, irresponsible that we don't put the burden upon ourselves to reach out with those results. After all, these people gave of themselves to us. I think it's the very least we can do to reciprocate their understanding.

I call on all my colleagues, especially my clinician scientist colleagues, to think rigorously, "How am I going to get the information back to these patients?" I think this has to happen prospectively, really at the time of trial activation and enrollment. Even if it's a positive study and it gets published in a highly reputable journal, often that article will be behind a paywall or it might only be presented at a conference with limited attendance.

It's important that we respect intellectual property, but it's even more important that we respect the fact that these patients are sacrificing something for us. We need to honor what they give.

This is Mark Lewis for Medscape. Thanks for listening.

Mark A. Lewis, MD, is director of gastrointestinal oncology at Intermountain Healthcare in Salt Lake City, Utah. He has an interest in neuroendocrine tumors, hereditary cancer syndromes, and patient-physician communication.

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