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Friday, January 20, 2023

3 Questions on… Whether Leukemia & Myeloma Trials Represent Patients at Risk

​With Jorge Cortes, MD, Director of the Georgia Cancer Center at Augusta University​

By Sarah DiGiulio

An important step in addressing disparities in care and outcomes for patients with cancer based on race is addressing disparities representative of patients in clinical trials. If studies investigating new drugs and other treatments for cancer do not accurately represent patients at risk of getting those cancers, that might affect whether the results of those trials are indeed accurate.

Researchers at Georgia Cancer Center at Augusta University set out to study the following: in pivotal clinical trials for drugs approved to treat leukemias and multiple myeloma, do the studies accurately represent the demographic and geographic diversity of populations affected by those cancers? The data were published in the Journal of Clinical Oncology (2022; doi: 10.1200/JCO.22.00504).

The researchers identified clinical trials from the FDA database that led to FDA approvals of drugs for these blood cancers. They found that a total of 41 (67.2%) trials leading to drug approval reported data on race and 20 (48.8%) on ethnicity. These trials included 13,731 patients in total. The data showed that Blacks, Native Americans, and Hispanics were significantly underrepresented in trials compared with the proportion in the general population affected by these cancers. Female patients were underrepresented in acute myeloid leukemia trials. Males were underrepresented in both multiple myeloma and chronic myeloid leukemia trials.

Asked whether the findings were surprising, study coauthor Jorge Cortes, MD, Director of the Georgia Cancer Center and the Cecil F. Whitaker, Jr., GRA Eminent Scholar Chair in Cancer, noted: “Perhaps not surprising, but disappointing precisely because we know about it, but we have not done much about it." He shared more about the findings with Oncology Times.

1. Why did you and your colleagues decide to pursue this research now?

“It is becoming increasingly evident that the populations studied in clinical trials are not fully representative of the populations in need. Health care disparities are a growing problem in our country, in general, and in cancer in particular. There are many steps needed to decrease these disparities. One of them is to ensure that clinical trials are available to and representative of all those in need.

“There has not been much done previously that is similar to our approach; what little has been done is mostly in solid tumors. The results are indeed aligned with what we report, that is, that studies are biased towards some populations by race, by sex, by ethnicity, and by the ability to reach a few major cancer centers."

2. What were the key findings from your research?

“Studies, particularly these pivotal trials, are typically concentrated in a few centers that are not accessible to many patients. In part because of this and in part because of the design and complexities of clinical trials, minority populations are significantly underrepresented even when the incidence of certain tumors is particularly common among them, such as multiple myeloma in African American patients. Another important finding is that many studies do not report clearly the population distribution among race and ethnicity. This should be an absolute requirement for reporting a study.

“The implications are that the results that we read in the literature and that lead to drug approval may not necessarily be extrapolated to all patients, particularly those who have different personal or social characteristics than those included in the trials. Studies should reflect the population in need and investigators and regulatory authorities should focus on this. To do this, we need to make sure we take the trials to the patients, not the patients to the trials."

3. What is the bottom line message you want cancer care providers to know about your work?

“We are looking at other tumors and also examining [the issues] from a global perspective, which is likely to further amplify the problem. Then we need to address how we solve the problem, as this is mostly a discovery approach. There is a need to expand the reach of clinical trials overall and particularly pivotal trials to make them accessible to all and representative of the general population. This is a central element, in my opinion, of starting to address health care disparities in cancer."