In cancer clinical trials, patients often overestimate benefit


I recently saw a patient who drove six hours to consult me. He was an ex-boxer, lightweight, and had leukemia that refused to clear up with a few rounds of chemotherapy. He sounded like an old movie version of the fighter Rocky, though his voice was raspy, like Burgess Meredith’s character, Mickey Goldmill, Rocky’s trainer.

“What should I do to get rid of my leukemia?” he told me earnestly. “I heard you had a study going on here. I want in!”

He was interested in signing up for a clinical trial of a new immunotherapy that harnesses a patient’s own immune system to target and kill leukemia cells. His doctor told him the trial is available at our cancer center.

The clinical trial was a Phase 1 study, more dramatically dubbed a “first in human” trial — the first time the drug was given to people after what’s called “preclinical” testing. The primary purpose of Phase 1 trials is to find the appropriate drug dose and to assess the safety of that drug – and Not To determine if the medication is actually working.

In fact, in these trials the chance that a drug will ever be effective in treating a patient’s cancer is low Historically less than 15 percent.

What motivates people to sign up for trials to receive an experimental drug before its efficacy—and even its dosage—is clearly established, and the purpose of such a trial appears to have nothing to do with shrinking a person’s cancer?

What motivates patients to enroll in clinical trials?

in one studySurveys asking this very question were sent to nearly 300 participants in the Cancer Trial. When patients were asked about their primary motivation for participating in a clinical trial, 45 percent of respondents who were enrolled in Phase 1 trials said they were motivated by the potential for medical benefits from the treatment.

The next highest motivations for adults participating in Phase 1 trials were to trust the study physician (17 percent), to maintain hope (15 percent), and to help future patients (4 percent). This last category is medical altruism, as in the desire to help advance medical science. These tend to be more common in patients with a better prognosis.

Why, then, is almost half of these patients enrolled in a cancer clinical trial with the hope of a successful drug, when the goal of the trial was only to find the right drug dose?

Imagine you are my patient, with leukemia that kept burning despite multiple rounds of chemotherapy. Your doctor tells you that there are no more treatment options available, so it’s best to go to a university hospital nearby, or to a large academic hospital where they may run a clinical trial for a new drug.

Communication gap in clinical trial enrollment

For someone like my patient, who feels well enough to drive six hours for a consultation, a clinical trial becomes the next therapeutic step, no matter what that trial entails or any promises that a drug might work, just because his doctor told him they exist. .

Perhaps the most common reason is that those of us clinical research participants do not effectively communicate the real goals of such early stage trials.

This ineffective communication can lead to therapeutic misconceptions—the belief that the purpose of the research is to directly benefit the individual patient enrolled in the trial, when in fact, only future patients will benefit from the research.

Another study It examined how clinicians communicated the risks and benefits of participating in a phase I trial of 85 families of pediatric cancer patients. The risks of drug treatments were discussed 95 percent of the time, with 81 families out of 85 involved. It’s a bit surprising that this didn’t happen 100 percent of the time these Phase 1 trials involved chemotherapy.

Treatment benefits were discussed nearly as frequently – 88 percent of the time, for 75 of the 85 families. Altruism was mentioned in 41 percent of the families. However, in 13% of the conversations, the clinical trial was actually described as a bridge to another treatment or to extend life — even though there was no evidence that these first-of-its-kind drug therapies might even shrink cancer.

“I’m not ready to throw in the towel yet.”

People with end-stage cancer pursue treatment for a variety of reasons, and are willing to put up with the overwhelming side effects of medication for the prospect of tiny benefits.

As healthcare providers, it is our responsibility to understand those drivers and to ensure that our patients do not enroll in a clinical trial with the wrong goal in mind – and to take special care not to mislead our patients about treatment goals. possible.

I asked my patient if he was sure he wanted to pursue another leukemia treatment, given the low chance of remission.

“Nah Duc, I’m not ready to throw in the towel yet. I’m still tough as nails!” He raised his arms and gave a few blows of air to emphasize his point.

He qualified for the trial and spent the next month in the hospital, enduring great side effects of the treatment but retaining his cool spirit throughout. But at the end of that month, the leukemia remained despite his and our efforts.

I saw him in my clinic one last time before he went home, and I apologized for how he had spent what would turn out to be his last weeks. I was horrified that he had spent this time in a foreign city, enduring the blows of experimental treatment, instead of being at home with his family.

waved to me. “Doctor, it was worth a shot, wasn’t it? And I think, maybe you’ve learned something with my studies, and I’m helping someone else in the future.”

His altruism was very impressive. And maybe for my patient, just getting the moxie to take that step of therapy — to get in the ring again and say he’s figured out all the avenues — was the goal all along.

Mikkael A. Sekeres, MD, MA is chief of the division of hematology at Sylvester Comprehensive Cancer Center, University of Miami. He is the past chair of the Food and Drug Administration’s Oncology Drugs Advisory Committee and author of the book Drugs and the Food and Drug Administration: Safety, efficacy, and public confidence. Follow him on Twitter @MikkaelSekeres.

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