January 31, 2020
3 min read

Few patients enroll in cancer clinical trials as first line of treatment

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Nicholas G. Zaorsky, MD,
Nicholas G. Zaorsky

An “exceedingly low” percentage of patients with cancer enroll in clinical trials as the first course of therapy, according to results of a study conducted at Penn State College of Medicine and published in Journal of the National Comprehensive Cancer Network.

“There just aren’t that many clinical trials available for patients when they’re first diagnosed with cancer,” Nicholas G. Zaorsky, MD, assistant professor of radiation oncology at Penn State College of Medicine, said in an interview with Healio.

“There may be trials available for patients whose disease comes back, but not as many for patients just diagnosed. For the trials that do exist, there are some preferences for the patients who will be enrolled,” he added.

Overall, less than 5% of eligible adults participate in cancer clinical trials.

In the retrospective cohort study, Zaorsky and colleagues gathered demographic and clinical information from the National Cancer Database on more than 12 million patients with 4 to 6 types of cancer between 2004 and 2015. They found that among these patients, only 11,577 (0.1%) participated in clinical trials as their first course of cancer therapy after diagnosis.

A greater proportion of trial participants vs. nonparticipants were white (88% vs. 84.8%), had metastatic disease (30.9% vs. 16.4%), had private/managed care insurance (56.4% vs. 41.8%) and had fewer comorbidities (Charlson-Deyo score 0, 81.9% vs. 75.7%). Univariate and stratified analyses showed patients with cancer who participated in clinical trials also had longer median OS (60 vs. 52.5 months; HR = 0.876; 95% CI, 0.845-0.907) than those not enrolled in a trial.

Zaorsky spoke with Healio about the need to make trials more inclusive, the importance of removing patient barriers and the lifesaving potential of clinical trial participation as a first intervention.

Question: How does the United States compare with other countries in clinical trial accrual?

Answer: Clinical trial accrual in the U.S. is very low among patients with cancer compared with other developed countries. There are some areas in Europe where, for certain cancers, accrual is in the teens, 20s or 30s. So overall, as a nation, I think we could be doing better.


Q: What patient populations are most likely to enroll in cancer clinical trials?

A: Our study found that young, healthy, white patients with private insurance and metastatic disease who received treatment at academic medical centers are more likely to enroll in clinical trials. Ideally, we want our clinical trials to be representative of the cancer population, and so we want to have minorities enrolled in trials. For example, we want to have patients not only at academic medical centers, but also community hospitals. We also don’t want trials to be limited to people who have private insurance.

Q: What are the some of the obstacles to participating in a trial that patients face?

A: When you tell a patient a trial is available, they might ask, “What’s in it for me? How do I even know if there’s a benefit?” There are often misunderstandings about being placed in a control group or a placebo arm. Often the placebo arm is the standard of care — these patients are basically getting what they would normally get — and the other arm is the experimental arm. It should be emphasized that whatever arm they are in, they are going to get the best care available. In fact, we found that patients enrolled in a clinical trial live longer than patients who are not enrolled in a trial. Being in the trial confers the biggest benefit in survival. The survival difference between the arms may be trivial. Also, many clinical trials may just have one arm.

Q: What should be done to increase clinical trial participation as a first line of treatment?

A: We don’t have focus enough on getting patients into clinical trials when they’re first diagnosed with cancer. Then, for the trials that are available, we must figure out how to get a more representative sample enrolled. I think there are different ways to go about addressing each problem. This research ties into quality of care, and we’re now looking at other quality metrics. We want to know, when a patient goes for cancer treatment, some of the factors associated with good quality care. We think that being on a clinical trial is one of those factors.” – by Jennifer Byrne

For more information:

Nicholas G. Zaorsky, MD, can be reached at 500 University Drive, Hershey, PA 17033; email: nzaorsky@pennstatehealth.psu.edu.


Zaorsky NG, et al. J Natl Compr Canc. Netw. 2019;doi:10.6004/jnccn.2019.7321.

Disclosure: Zaorsky reports no relevant financial disclosures.