‘Substantial’ contributions: More than 20% of patients participate in cancer research
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Key takeaways:
- Participation in cancer treatment trials has more than doubled over the past 30 years.
- Patients also contribute to other trials, including biorepository, registry, genetic and quality of life.
More than 20% of patients with cancer participate in some form of clinical study, and that number is considered a “floor” estimate, according to study results published in Journal of Clinical Oncology.
Involvement in treatment trials has more than doubled in the past 3 decades, and individuals have also shown interest in other research, including biorepository, registry, genetic and quality-of-life studies.
“The low participation of adult patients with cancer in clinical trials has been obviously a concern over many decades,” Joseph M. Unger, PhD, MS, associate professor at Fred Hutch Cancer Center, told Healio.
“It really does drive how fast clinical research can be done and how fast new treatments can be discovered,” he added. “A lot of that, intentionally or otherwise, has been placed at the feet of patients themselves as somehow not contributing in a way that they should. We’re trying to take the onus off the patient and put it on the system and the structural barriers that get in the way of patients participating in research.”
Roughly 2% to 3% of individuals with cancer participated in treatment trials in the 1990s and early 2000s, according to background information provided by researchers.
Estimates have remained low for years, but Unger and colleagues long suspected those percentages to be misleading due to outdated data.
A contemporary analysis
Unger has been part of multiple investigations into clinical trial participation, including one published in Journal of the National Cancer Institute in 2021, which found 55% of nearly 10,000 patients agreed to partake in a trial when offered.
“The problem is that it's just hard to get access to good national data,” he said.
The American Cancer Society Cancer Action Network found a new approach in a collaboration with the Commission on Cancer (CoC).
They used the CoC accreditation program, which includes 1,200 programs and 3,600 sites that encompass more than 70% of cancer diagnoses in the U.S. annually, to evaluate data between 2013 and 2017.
Researchers found 7.1% of patients participated in treatment, 12.9% in biorepository, 7.3% in registry, 3.6% in genetic, 2.8% in quality-of-life, 2.5% in diagnostic and 2.4% in economic studies.
In total, 21.9% of individuals joined a trial.
“The 7% estimate — we had a suspicion from our prior paper that it would be higher [than the 3%]. It kind of affirmed that,” Unger said. “But the one in five patients who participate in trials — any kind of trials — was surprising for sure. In our view, that’s a floor. It’s at least one in five. It's probably higher.”
Unger expressed optimism around the nontreatment trial results too.
“The biologic and genetic studies — what’s really important there is that the paradigm of cancer treatment has shifted to more precision-oriented medicine,” he said. “I was encouraged to see participation contributions to biorepositories to be as high as it was. That’s really critical for the new treatment era.
“Quality-of-life studies alongside treatment trials used to be somewhat of a niche effort,” he continued. “It took a lot of hand holding to do those types of studies and get the questionnaires administered appropriately. That has all changed in the last decade.”
The data Unger and colleagues collected are from 2013 to 2017, which translates into a 7-yer time lag despite being the most recent known evaluation of trial participation.
“Since 2015, the inclusion of quality-of-life studies in phase 3 trials is almost ubiquitous,” he said. “That’s why you see the quality-of-life estimate less than half of the treatment trial. It’s partly because of that time lag. I suspect if you were to have access to data starting at 2020 it would be much higher.”
‘It’s the system’
Unger said the reason percentages are not higher has more to do with barriers, not interest.
In a 2019 study in Journal of the National Cancer Institute, Unger and colleagues found that more than 50% of individuals do not have trials available near them, and more than 20% did not meet eligibility criteria.
“After you remove those three out of four patients, then you’re finally down to where the physician has a discussion with their patient,” Unger said. “The universe of opportunities for patients to participate is much smaller than is usually understood. It’s not 100%. It’s really like 25%, or even less. That’s not on the patient, and it’s not on the physician either — it’s the system. It’s hard to nudge the needle in a substantial way, either through patient or physician actions, when you’ve reduced the pool of opportunity so much right at the beginning.”
Unger would like to continue to examine CoC data to continually update participation estimates.
He said the COVID-19 pandemic made inclusion in trials easier due to policy changes, and he suspects many of those policies to remain “permanent” fixtures.
“For instance, allowing patients to consent to a trial remotely instead of having to come to a clinic — that’s a big deal,” Unger said. “Allowing local physicians to administer some aspects of protocol therapy — that’s a big deal. I’m chairing a working task force that’s sponsored by ASCO and the Friends of Cancer Research where we’re examining some data from the time of trial conduct during the pandemic to see whether these flexibilities that were introduced by the NCI and the FDA during the height of the pandemic actually harm data quality.”
Eligibility criteria and decentralization of trials continue to be barriers, although multiple organizations have worked to address those issues.
Treatment trial participation continues to happen far more at NCI-designated comprehensive centers (21.6%) than other locations, such as non-NCI academic programs (5.4%), integrated network programs (5.7%) and community programs (4.1%), researchers found.
However, the NCI has made “substantial” investments into community programs, Unger said.
“About one-third of all enrollments to NCI-sponsored trials are from those community, minority, underserved sites,” he added.
Community programs need more investment, however, to expand their impact.
“I think pharma increasingly is aware that they need to make their trials more accessible, in part because of the representation issue,” Unger said. “There’s this guidance that was put out by the FDA that says you have to have a plan to make your trials more representative for industry sponsors. I think one of the ways that they’re seeking to address that is by building out their programs beyond just the large academic centers. The investment comes from both federal and industry. I think federal investment has been fairly good in that respect. It’s really industry that has not expanded out beyond the large academic centers in a way that makes trials more accessible.”
Still, participation has improved, and Unger believes it will continue to do so.
“Policies around cancer clinical research are really driven by having a clear understanding of how commonly patients participate,” Unger said.
“If you know that few patients with cancer participate in clinical research, then that informs how you interpret your trials. It informs what kind of policies you implement to improve access to trials,” he added. “The patients are contributing in a very substantial way.”
References:
- Unger JM, et al. J Clinl Oncol. 2024;doi:10.1200/JCO.23.01030.
- Unger JM, et al. J Natl Cancer Inst. 2021;doi:10.1093/jnci/djaa155.
- Unger JM, et al. J Natl Cancer Inst. 2019;doi:10.1093/jnci/djy221.
For more information:
Joseph M. Unger, PhD, MS, can be reached at junger@fredhutch.org.
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