Women and Minority Populations in Clinical Trials

Medical researchers have been required by law to include women and representatives of minority groups in clinical trials since the early 1990s, but the impact of these requirements on health outcomes for these populations is unclear, a panel of experts concluded at a symposium on April 24 at the Dana-Farber Cancer Institute.

The event, Women and Minority Enrollment Targets in Clinical Trials: A Symposium and Panel Discussion on Diversity in Medical Research, was co-sponsored by the HSPH Bioinformatics Core as well as the DFCI/Harvard Cancer Center Cancer Disparities Program-in-Development, and several others.

"Inclusion is a good thing, no question, and the intent behind these policies is very much on target, because we can't be studying just one group in society," said keynote speaker Steven Epstein, professor of sociology at the University of California-San Diego and the author of Inclusion: The Politics of Difference in Medical Research.

Before these requirements were enacted, clinical trials were often populated by middle-aged white men, he noted.

"Everyone in principle deserves the benefit of all the resources we devote to research," he added. "The question is what are we doing with the data that we are generating, and are we asking the right kind of research questions to actually figure out why we have such inequalities in the domain of health in our society."

Epstein noted that the inclusion requirements resulted from the prodding by female members of the U.S. Congress and female scientists at the National Institutes of Health back in the 1980s and 1990s.

Opponents dismissed the idea at the time as bad science or political correctness, he noted. Others said that such inclusion would be too expensive.

David Williams, Florence Sprague Norman and Laura Smart Norman Professor of Public Health at HSPH, said that simply including underrepresented groups in studies is not enough.

"Inclusion is important, but we need to identify better markers than race [and gender] to identify the potential contribution of biological and genetic factors," he said.

Social class, Williams said, is a stronger prediction of variations in health than race, ethnicity, or gender. "Where you live and work has a lot more to do with your health than your genetics, especially in terms of explaining group differences," he added.

Lee Nadler, senior vice president of experimental medicine at DFCI, predicted that the importance of race and gender as factors in health outcomes will lessen as the examination of the human genome leads to more personalized medicine, including therapies that target specific genes.

Such targeted therapies will take some time to develop, said Worta McCaskill-Stevens, program director, Community Oncology and Prevention Trials Research Group, National Cancer Institute. This type of individualized targeted therapy is not ready for prime time, she said. In the meantime, inclusion in clinical trials by race and gender remains important. It is important for accountability of grantees in providing clinical research options to all populations, as a resource for relevant disciplines to address health disparities along with the clinical questions, and for tissue collection to support exploratory hypotheses for individualized therapies.

Steven Joffe, pediatric hematologist/oncologist and ethicist at DFCI, said that the historical underrepresentation of women and minorities in clinical trials raises questions of justice both for individuals and for groups. He was less concerned about individual justice, noting there is no good evidence that patients are disadvantaged by having reduced access to clinical trials.

"We don't have evidence to prove that patients in clinical trials are better off than those being treated outside a trial," he said. On the other hand, he concurred with other panelists that social justice requires the inclusion of diverse groups in clinical trials, so that data from the trials will be broadly generalizable.

Jerry Avorn, professor of medicine at Harvard Medical School and chief of the Division of Pharmaco-epidemiology and Pharmaco-economics at Brigham and Women's Hospital, said that a big problem not being addressed is follow-up after trials are over, when drugs are approved and on the market.

In general, the requirements to include women and representatives of minority groups — and more recently children — in clinical trials have at least caused people in the field to consider the problem of disparities, Epstein concluded. "They make us think, and there is tremendous virtue in that," he said. 

—Michael Lasalandra. Photo by Suzanne Camarata.