Diversity in Phase I Metastatic Cancer Drug Trials Declined From 2000 to 2018
Participation among American Indian or Alaska Native, Asian or Pacific Islander, Black, and Hispanic or Latin American people in phase I clinical trials for drugs that treat metastatic cancer decreased from 2000 to 2018, according to a study.
The study was published in the Nov. 3, 2022, issue of the journal JAMA Network Open. Read “Participation of Patients From Racial and Ethnic Minority Groups in Phase 1 Early Cancer Drug Development Trials in the US, 2000-2018.”
About racial diversity in clinical trials
Clinical trials are carefully designed research studies that depend on volunteers to see whether treatments can improve medical outcomes.
Clinical trials looking at new treatments are usually conducted in a series of four steps, or phases, from phase I to phase IV. Each phase builds on the one before it:
Phase I trials look at the best way to give a new treatment, as well as its safety, side effects, best dose, and timing.
Phase II trials look at how effective the new treatment is.
Phase III trials compare the safety and effectiveness of the new treatment with the current standard of care.
Phase IV trials look at whether the new treatment has benefits or long-term side effects that researchers did not see in the phase II and phase III trials.
Clinical trials are the main way scientists develop new and improved cancer treatments. Yet studies show that less than 5% of adults diagnosed with cancer participate in clinical trials, and most of those participants are white.
Black Americans make up more than 13% of the U.S. population. But in the clinical trials that led the U.S. Food and Drug Administration to approve four new breast cancer treatments in 2020, only 2% to 9% of participants were Black. Similarly, Hispanic or Latin Americans make up 18.5% of the U.S. population, but accounted for 0% to 9% of participants in those same trials.
Researchers have found a number of barriers that stop people of color from participating in clinical trials, including:
travel and transportation time and expense
out-of-pocket costs
lack of trust in the healthcare system
lack of awareness about clinical trial opportunities, possibly because doctors don’t discuss clinical trials with them
If researchers don’t test new cancer drugs in a group of people that represents the general population of people diagnosed with cancer, they can’t assume the drugs are safe and effective for people of different races and ethnicities.
About the study
Much of the research looking at diversity in cancer clinical trials has looked at phase III trials. In this study, the researchers wanted to look at phase I drug development trials.
The researchers looked at information from 221 phase I studies for metastatic cancer drugs from 2000 to 2018.
The researchers compared the races and ethnicities of the people in the studies with the rates of cancer in various races and ethnicities in the North American Association of Central Cancer Registries’ Cancer in North America database. The North American Association of Central Cancer Registries is an umbrella organization for cancer registries, governmental agencies, professional associations, and private groups in North America interested in enhancing the quality and use of cancer registry data. All central cancer registries in the United States and Canada are members.
From 2000 to 2018, compared with the overall number of people diagnosed with metastatic cancer, the results showed that white people were overrepresented and several other racial and ethnic groups were underrepresented in phase I trials studying metastatic cancer drugs.
The researchers looked at rates of trial participation versus actual diagnosis rates and found these differences:
86.1% versus 84.1% for white people
4.5% versus 5.9% for Asian or Pacific Islander people
6.2% versus 11.8% for Black people
The researchers looked at information from 2000 to 2011 and information from 2012 to 2018 and found an increase in overrepresentation by white people. There also was a corresponding increase in underrepresentation in the clinical trials by American Indian or Alaska Native people, Asian or Pacific Islander people, Black people and Hispanic or Latin American people.
Rates of trial participation versus actual diagnosis rates in 2000 to 2011 versus 2012 to 2018:
increased by 2.7 percentage points for white people
decreased by 0.12 percentage points for American Indian or Alaska Native people
decreased by 1.75 percentage points for Asian or Pacific Islander people
decreased by 2.9 percentage points for Black people
decreased by 3.2 percentage points for Hispanic or Latin American people
“This study highlights disparities in the enrollment of patients from racial and ethnic minority groups in phase 1 drug trials for metastatic cancer; this gap appears to be worsening over time,” the researchers wrote. “Phase 1 cancer trials are often conducted at academic institutions because the logistics of running early drug trials make them difficult to conduct in community settings. Because patients from racial and ethnic minority groups may be less likely to receive care at academic medical centers, this could explain accrual disparities. Clinical innovations, such as hospital-at-home initiatives and establishing community-academic alliances, may lead to higher trial enrollment in community-based partner sites and expand access to phase 1 trial drugs to patients with cancer who may not regularly obtain care at academic medical centers. Using telemedicine platforms to offer virtual visits when available, establishing direct-to-patient shipping for oral therapeutics, and forming partnerships between advocacy groups, trial sponsors, and research teams to expedite accrual to studies may also help lessen disparities.”
What this means for you
Because of a lack of diversity among clinical trial participants, studies are testing new cancer treatments in groups of people that are not representative of the broader population that are going to receive these treatments if they are approved.
“We need everyone appropriately represented because we want to make sure a therapy works the same way and the side effects are the same in different populations,” Karen Winkfield, MD, PhD, a radiation oncologist and executive director of the Meharry-Vanderbilt Alliance in Nashville, told Breastcancer.org. “We also want to make sure we are providing precision medicine. Sometimes a medicine might work better in one population than another. We need to learn these things up front so we can give each patient the right medicine at the right time.”
Being part of a clinical trial means you’re helping find researchers better treatments. Your participation could help save lives. Still, it’s a very personal decision.
If you’re interested in finding a clinical trial that might be right for you and your unique situation, ask your medical team. If your doctors aren’t knowledgeable about clinical trials, ask if they can refer you to a doctor who is. You also can search ClinicalTrials.gov to see which trials you might be eligible for.
Read our Special Report: Increasing Racial Diversity in Breast Cancer Clinical Trials for more information.
— Last updated on January 25, 2023 at 6:13 PM