Sugar Pill Myth: What Am I Really Getting in a Clinical Trial?

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Topics include: Treatment | General , Treatment and Understanding

A barrier that deters patients from enrolling in clinical trials is the belief that they might receive a sugar pill and get no treatment at all. Clinical Trial MythBusters debunks this myth with Dr. Heather Wakelee, Director of the Stanford Cancer Clinical Trials Office at the Stanford University School of Medicine, to find out what kind of care patients really undergo when participating. Dr. Wakelee also explains how patient involvement in trials affects the advancement of cancer research.

The Clinical Trial MythBusters series is a Patient Empowerment Network program produced by Patient Power. We thank Astellas, Amgen and AbbVie for their support.

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Please remember the opinions expressed on Patient Power are not necessarily the views of our sponsors, contributors, partners or Patient Power. Our discussions are not a substitute for seeking medical advice or care from your own doctor. That’s how you’ll get care that’s most appropriate for you.

Andrew Schorr:

So, Dr. Wakelee, I have—we discuss this myth every time, but I think we have to do it. And that is, people in cancer trials worry that they’re, if you will, not gonna get the good stuff, or the promising stuff, that they—in other conditions, that they’re gonna get a sugar pill; “This could be lifesaving for me, and oh my God, I find out later, there was nothing in it.” So help us understand how you operate at Stanford related to people getting quality care.

Dr. Wakelee:    

Well, I think that’s a really important myth with a trial. There are no trials that have patients now randomized to get something that has no chance of working. The comparative trials are always looking at something that is known to be effective—the quote-unquote “standard,” which is the same thing that someone would get if they weren’t on a trial, versus getting the newer drug.

And it’s really critical, as much as we all have hope and faith in the trials, and every treatment that we know of today is there because of patients who were willing to participate in earlier trials. There are a lot of medications that we worked to develop that don’t help people, that are not better, and something that’s new always sounds more promising than something that’s old. Because when something’s been around a while, you know it, and you know the good and bad of it, and when something’s new, you only really have the promise, and that doesn’t claim as much of the bad potential.

And so, it’s that balance, and the reason that we have to do the trials is that some of the drugs, despite how well they work in all of the earlier models and laboratory investigations, they don’t always work in people. And we don’t know that if we don’t try them, and so I think that’s one of the things that is always difficult, because of course, I couldn’t be an oncologist if I wasn’t an eternal optimist and hoping for all of my patients that they’re gonna do well. But having done this a while, I’ve also seen a lot of situations where the treatments that we think are gonna be the best don’t work, and that’s why the trials are so important.

If we take everything that sounds great out of the lab and give it to people, we actually might be harming a lot of folks. And so the studies that compare one treatment versus another remain an essential part of what we do, and I look back on—when to a patient who’s having a hard time with that concept, I think of a few specific examples, and…

…I work in lung cancer, and one of the greatest things that have happened in lung cancer treatment over the last 15 years was understanding that some lung cancers are different than others. There are these particular subtypes, where we have mutations that happen in a gene in a cell and that cell then is what develops into the cancer. So the ones many people have heard about are, like EGFR and there’s ALK.

And I’m not gonna go into all the specifics, but when we first learned about that, we learned about medications that hit EGFR. And there are particular pill drugs that work there, and there was also chemo that we also knew worked. And so when we were first learning about EGFR and the pill drugs that work there, everyone assumed, well, if you just added that to the chemo, it was gonna be great. And people were gonna do so much better if you could combine them. It just made sense.

And so, there were studies where patients got just chemo, or they got chemo plus the EGFR drug. And the people on those studies who were randomized to get just the chemo, you know, they felt like they were being cheated, like they weren’t getting the best thing. But when the studies were all done and we looked at the data, we realized that actually it didn’t make any difference. It just added toxicity.

And so, that’s one key example that I can think of, and there was a placebo, but it wasn’t that patients were not getting any treatment. They were either getting what they would get if they weren’t on the trial, by getting the chemo, or they were getting chemo plus these other pills. And I use that as one example, but there are a lot of those I’ve seen, where people are either getting what’s standard, or they’re getting what’s standard, plus. And the standard, plus, is not always better.

Another example in that same world, that same—of EGFR, where now we know that if someone does have one of those mutations in their tumor, they start taking the pill drug, that’s the first thing they do. Not chemo, yet. And there was this idea, well, if you start on that, and then when the cancer becomes resistant, and you have to stop taking the pill. We traditionally would switch to chemo, but there are a lot of people who believed, well, if this EGFR drug’s working and it stops working a little, and then you add chemo to it then, that’s gonna be better. And that was what was done, but trials were developed to ask that question. So people either stayed on their EGFR drug and started chemo, or they stopped it and started chemo, and again, people thought they knew the answer.

They were quite sure that it was much better to keep going, but the studies were done, and it was actually worse to do it that way. But we would never have known that if we hadn’t done the trial and if people weren’t willing to be randomized and told, “I know you think it’s better to take the pill, but in this case it isn’t.” And that’s why it’s so important that we still do these trials and that people understand if we really honestly absolutely know the truth, then we don’t do the study.

But a lot of times we only think we know the truth, and until we do the trial, we don’t. And so, it’s so critical, and that’s why it’s so wonderful that you’re doing this program and helping people to understand, because there’s so much fear, “Well, I’m not gonna get the good stuff.” But the truth is we don’t know what’s good until the studies are done.

Please remember the opinions expressed on Patient Power are not necessarily the views of our sponsors, contributors, partners or Patient Power. Our discussions are not a substitute for seeking medical advice or care from your own doctor. That’s how you’ll get care that’s most appropriate for you.

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Page last updated on November 6, 2017