By: CAPT Richardae Araojo, Pharm.D., M.S., Associate Commissioner for Minority Health, Office of Minority Health and Health Equity; Ann T. Farrell, M.D., Director of the Division of Hematology Products within the Center for Drug Evaluation and Research; and Jovonni Spinner, M.P.H., CHES, Senior Public Health Advisor, Office of Minority Health and Health Equity
As Minority Health Month comes to a close, the U.S. Food and Drug Administration (FDA) continues to work on solutions to eliminate health disparities among diverse racial and ethnic populations. The disparities in health span everything from cancer and diabetes to heart disease, sickle cell disease, and HIV/AIDS. The FDA’s work on these issues is a priority—and is intrinsic to the work we do across the agency.
The FDA, including the Office of Minority Health and Health Equity (OMHHE), promotes and protects the health of diverse populations and strives for health equity for all, including those who experience disparities related to race and ethnicity.
One example is the agency’s work to encourage diversity in clinical trials. Racial and ethnic minority populations continue to be underrepresented in clinical trials and remain disproportionately burdened by many chronic and debilitating diseases. This is due to factors including a lack of trust in the medical system—in part due to historical mistreatment—as well as a lack of transportation, time, or knowledge about clinical research opportunities.
The FDA’s primary role in clinical trials involves reviewing clinical research as part of the regulatory process. As an agency, the expectation is for medical product research to ensure ethical and legal protections for patients and to reflect the relevant science. To this point, FDA regulations require developers who submit applications for medical products to analyze clinical trial data by sex, age, and race. And the FDA continues to regularly work with stakeholders—including federal partners, medical product manufacturers, medical professionals, and health advocates—to encourage diversity and inclusion in clinical trials. To this end, the agency has issued guidance on the collection of race and ethnicity data in clinical trials. To continue efforts to communicate with and educate patients, the FDA created a Minorities in Clinical Trials Initiative that includes patient videos, with additional materials planned for this year. The agency continues to engage with patients to increase understanding and awareness of the benefits and risks of clinical research and the importance of having the facts when making decisions about medical care.
The FDA also has made strides related to disease and treatment areas for which there are noted health disparities. One example of this is the agency’s work to support research focused on minority health. The FDA’s work on sickle cell disease, a genetic condition that affects the body’s red blood cells, is another example. Sickle cell disease, also a public health priority for the U.S. Department of Health and Human Services (HHS), is most common among African Americans. But other racial and ethnic groups also are affected, including Latinos and people of Middle Eastern, Indian, Asian, and Mediterranean backgrounds. And as HHS remains committed to breaking down barriers to treatment and finding new cures for patients with sickle cell disease, the Administration also has committed to supporting research to develop a cure for the disease and has noted that clinical trials to accelerate the development of new gene and cell-based therapies will be conducted as part of the National Institutes of Health’s Cure Sickle Cell Initiative.
Notably, the FDA approved two new treatments in 2017 for sickle cell disease, one for adults and children age 5 and older, and a second for children age 2 and older. These medical products were needed options for patients who hadn’t seen movement in this area for about two decades. But as patients around the country and the world continue to deal with the effects of this debilitating condition, the FDA understands that more needs to be done.
The FDA is collaborating with patients, academics, and the pharmaceutical industry to encourage the development of new treatments for sickle cell disease (SCD). Watch this video, and read the Consumer Update to learn more.
To encourage industry’s development of even more treatments—and an eventual cure—for sickle cell disease, staff at various FDA Centers and Offices have coordinated to discuss next steps, new treatment options, and the best ways to communicate about known information.
For instance, the FDA’s Center for Drug Evaluation and Research (CDER), not only reviewed and approved the two new treatments but is actively encouraging companies to develop additional therapies. Today, a growing number of new, potential therapies are now in the pipeline, with products now in early- and late-stage clinical trials. Companies can request, and the FDA can grant, expedited and priority review to therapies intended to treat this rare disease—including via “fast track” and “breakthrough therapy” designation, which allows companies to have early and more frequent interactions with the agency.
In October 2018, the FDA led a joint public workshop with the American Society for Hematology to discuss clinical trial endpoints for sickle cell disease. FDA staff included participants from the Oncology Center of Excellence and Centers and Offices from across the agency. This workshop, which included well-attended panels on biomarkers, patient-reported outcomes, and pain, sought to bring uniformity and standards to existing disease endpoints, identify gaps, and propose development of new endpoints as a focus for future research. FDA Offices and Centers are now working on identifying the best ways to disseminate information about these activities so that patients, stakeholders, and government partners can continue to lend their voices to these multi-pronged efforts while being cognizant of the agency’s activities. Additional meetings to focus on endpoint development are planned for later this year.
Whether the focus is on clinical trials more broadly, sickle cell disease, or other diseases and conditions for which health disparities still exist, the FDA is an active participant in these national efforts. Differences in health outcomes continue to exist, but as we spotlight them and work to develop timely and forward-looking solutions, including more diverse representation in clinical trials as well as timely and impactful communications with our various audiences and partners, the FDA is doing its part to both recognize and reduce them. Today and every day, the FDA pledges to continue this important work with stakeholders, other local and federal agencies, and patients across the country.