When the Food and Drug Administration (FDA) recently gave a presentation on diversity in clinical trials, Dr. Ricardo Rojo wasn’t surprised to hear that — at least for medicines where gender is not overtly relevant — research participants today are predominantly white men.
Rojo, Pfizer’s first Global Lead for Diversity in Clinical Trials, is on a mission to change this. For years, the FDA, the pharmaceutical industry and various stakeholders have been working to improve racial, ethnic and gender representation in clinical research, which is essential to determining the safety and efficacy of new medicines. “When we include members of communities of color, it not only better informs us about a therapy and its potential side effects, it also helps patients become more knowledgeable about their conditions and take better care of their health. These patients become beacons for others in their community, educating them and drawing more participation in clinical trials,” says Rojo, who is based in Pfizer’s Groton, Connecticut, research site.
Clinical trials are the first time a new medicine, which has undergone years of lab study, is tested in humans. Generally, testing begins with a small group of healthy volunteers to assess the drug’s safety (Phase 1 trials), and then proceeds to patients who have the disease or condition to test its efficacy and side effects (Phase 2). Most often there is a final phase, in which a large group of patients potentially numbering in the thousands, is enrolled to confirm the efficacy of the new therapy and compare it with the current standard treatments (Phase 3).
It’s important to conduct these studies in participants from a variety of backgrounds, because people respond to medicines differently depending on gender, race, age and genetics. To better address the health needs of all groups, we need data that better reflect the true demographic makeup of the U.S.
Rojo, a physician and neuroscientist, is leading a team to implement a variety of tools and initiatives to increase diversity in human studies. Read on to learn how ride-share drivers, babysitters and community health workers are part of these efforts.
Get Science: Can you explain why diversity matters in clinical research?
Diseases are often caused by an interplay of biological, environmental and genetic factors, and we need to gain insights into this complex picture. We know, for example, that African-Americans have a higher risk for diabetes and cardiovascular diseases and certain cancers, and that Asian-Americans have higher rates of hepatitis B. Genetic variations play a large role in how we respond to medicines. These genetic differences can make a drug more or less effective or safe in one group versus another, and obtaining more data will help us try to ensure that medicines are safe and beneficial to all patients.
GS: How are clinical trial participants recruited?
RR: It depends on the disease area. In cancer, for example, if the standard therapies are not working, your physician could recommend a trial as an immediate option. It really depends on the physician you go to and their awareness of drugs in development that could be potential therapies for their patients. Most of the time clinical trials are discussed between physicians and patients, There are several ways in which Pfizer is facilitating clinical trial recruitment so that the right patients for a potential therapy are matched with the right trial.
GS: What are some of the reasons why clinical trials are so lacking in diversity?
RR: Two barriers to clinical trial participation among minorities are lack of awareness and trust. Some believe the mistrust stems from historical events such as the Tuskegee syphilis trial from 1932 to 1972. In that study, a huge injustice was inflicted on a group of African-American subjects whose syphilis went untreated without their knowledge. Some believe that history has contributed to mistrust in clinical research overall among African-Americans. A lack of health literacy is another contributing factor.
GS: What is the FDA doing to address this issue?
RR: There are no minority participation quotas mandated by the FDA, but it has been doing a lot to improve reporting and transparency in the research process. It used to be required to report on only four racial groups: white, black, Asian, or other. Self-reporting was recently updated to include additional subgroups and multiple races. An obvious example is Barack Obama, who had a Caucasian mother and African father. Previously he would have only had the option to choose white, black or other, whereas now he can choose both white and black. Additionally, the FDA launched a “Drug Trials Snapshots” website that allows patients taking a prescribed drug to look up how their racial, age or gender group responded to that treatment during clinical trials.
GS: What are some of the initiatives that Pfizer is working on to improve minority participation?
RR: We have developed a set of videos for doctor’s offices and hospital waiting areas to explain what a clinical trial is in very plain language, because we know that lack of health literacy is a core factor leading to lack of participation in clinical research. It would also help to educate patients about the variety of ethical guidelines and policies in place today to protect their rights.
In addition, we’re helping to raise awareness among staff at research sites and hospitals.
We want to create the infrastructure to make including subjects from specific racial groups a seamless process. Through a variety of workshops we can offer them recruitment strategies that work. We’re developing tools to help those who enroll patients in trials consider diversity at the earliest possible opportunity.
Then there’s the logistical challenges that every patient faces. We know how complicated and time-consuming it can be to participate in research. It’s not like when you go see a doctor every six months. Sometimes visits are very long and way more frequent. As a result, we are taking steps to make it easier to participate.
Participants are often reimbursed for their expenses on a monthly basis, and that can be an issue for people who are socioeconomically disadvantaged. We’re working with the Clinical Trial Innovation group on reducing expenses through a collaboration with ride-sharing services that will pick up patients at their door and deliver them to the site. Afterwards, they’ll take them from the visit to their workplace or back home. We’re also making it easier for participants who need child care during their visit. Our shared goal is to make the access part of clinical trials a non-issue.
GS: You’ve spent a good portion of your career addressing health care disparities. How does research participation tie in to that larger issue?
RR: Drug development is centered around knowledge. The more we know about different groups of patients, the more we can realize the goals of precision medicine to make drugs that are relevant to all. For instance, including members of communities of color not only better informs us, it puts those patients in a better position because they know much more about their disease and are more adherent to treatment. They basically become better patients—and that’s another way of combating health disparities.
If enough members of a given ethnic community participate, the lack of diversity in clinical trials should become less of an issue, and we may be able to develop drugs that work across broader populations. There’s a lot we don’t know right now about how various groups respond to drugs. But by working with the various stakeholders— such as patients, community leaders, research centers and regulators— I’m optimistic we’ll fill this void.