Getting treatment for a rare disease is already challenging, but the complex challenges are compounded for minorities with rare diseases. Inequalities in access to healthcare amongst racial and ethnic minorities is well-documented and has resulted in multiple efforts by the Food and Drug Administration (FDA) to drive diversity and inclusion in clinical research.
I recently attended a discussion panel during the MassBio Rare Disease Day titled “Addressing Health Inequality in Rare Disease Research.” The framework for the panel was to examine the current state of rare disease research and how participation of minorities with rare diseases in clinical trials may be improved. The panel resonated with me for two reasons: 1) I have been working in clinical research for many years; 2) I am a minority woman.
As a minority, I am intimately aware of the stigma associated with clinical trials within the community. There is a strong mistrust felt by many minorities that is rooted in history, folklore, and lack of exposure to factual knowledge. A notable source of the mistrust is the Tuskegee experiment that resulted in young black males with syphilis going untreated, though a cure existed. This is just one example of an instance where a minority population was exploited for the advancement of science. So how does one go about changing people’s mindset? Even more, how do we make progress together?
As noted by one of the panelists, the researchers, biopharma companies, and advocacy groups must “speak to people where they are.” Those that are in the position to change the negative perceptions must make the effort to educate by acknowledging the mistrust, fears, and history that have affected minorities, and then through the patients’ lens, offer relatable and appropriate information.
Now I want to take this “speak to people where they are” mindset a bit further so that our industry has talking points with their colleagues when they’re addressing how to incorporate minorities in their clinical study design.
The Importance of Minorities in Clinical Research
The participation of racial and ethnic minorities in clinical research, especially for rare diseases, is vital to the advancement of knowledge about the treatment of these diseases. Take for instance sickle cell disease, a group of blood disorders that are normally inherited, the most common of which is sickle cell anemia. Sickle cell anemia is rare in the United States but affects African Americans at a greater incidence. One in every 365 African American children will have sickle cell anemia (CDC, 2016). Through a search on Clinicaltrial.gov, 89 clinical trials for this disease in the United States have been terminated, suspended, or withdrawn due to poor enrollment. Controlled research has been conducted to try and understand the barriers to participation.
An example is the study conducted by Lamia P. Barakat et al, published in the American Journal of Preventative Medicine (2016 Jul; 51(1 Suppl 1): S78–S86). This study evaluated proximal (age, gender, disease severity, perceived stress, SES) and distal (religious beliefs, social support, instrumental support) factors related to mistrust of research among caregivers of children with sickle cell disease (SCD) and adolescents and young adults (YAs) with SCD. The interesting takeaway from this article was that, for caregivers, the barriers to participating were perceived stress and religious beliefs, while for YAs, the mistrust appeared to be gender driven. This article is just one example of research that has been conducted that can help guide discussion and practices toward diversity inclusion within clinical research.
Lots of Content on Diversity in Clinical Trials, but it Loses the Patient
There have been many conferences and webinars in the past few months focusing on diversity and inclusion in clinical research. In the conference and webinars that I have attended, I do not see that these issues noted above are at the forefront of the discussions.
It is great, however, that the issue of inclusion has been at the forefront primarily due to the recent pandemic. However, these conference and webinars are geared toward those running the clinical research and not toward the trial’s participants. There is more that needs to be done to “speak to people where they are” to foster trust within communities. And trust in minority communities is essential.
Let’s Bring the Patient Back into Clinical Trial Design
For instance, these conferences and webinars should be advertised toward patient advocacy groups, community groups, schools, or church groups to meet the patients where they are. As researchers, what can we do to educate not just the potential participants, but also researchers? Here are a few items to consider when planning your next clinical trial to ensure a diverse representation: