Tokenism In Clinical Trials For Rare Disease Patients
By Jenifer Ngo Waldrop, executive director, Rare Disease Diversity Coalition (RDDC)
7,000 unique diseases defined as rare, only 10% have FDA-approved treatments. For those also navigating the added hurdles of systemic discrimination and racism in healthcare, hope is often completely out of sight.
Patients who find themselves at the intersection of rare diseases and marginalized populations are at critical risk, and our healthcare system is ill-equipped to support them. The FDA’s recently unveiled guidance for increasing diversity in clinical trials has been hailed as a pivotal step toward addressing the systemic challenges facing this underrepresented community. Yet, the absence of accompanying penalties or incentives for companies running clinical trials has set the stage for a lackluster rollout that will inevitably fail to enact true change for those most at risk. It is imperative that we broadcast the urgent nature of this crisis and implement mandated policies designed to expand diverse representation in clinical trials without negatively impacting communities of color.
Rare diseases are more common than one might think. In fact, more than 30 million Americans — nearly one in 10 — are estimated to have a rare disease. Despite the large population impacted, support and treatment options are incredibly limited. By some approximations, it can take an average of five years and consultations with more than seven clinicians to reach an accurate diagnosis for a rare disease.
This bleak reality is even worse for patients of color with rare diseases. This community experiences a disproportionate amount of hardship due to the overwhelming intricacies of rare disease journeys, which encompass severe ailments such as lupus, sarcoidosis, sickle cell anemia, and thalassemia. They face widespread disparities in access to care and are all too often stymied by a lack of insurance coverage or access to transportation. They must also overcome deep-rooted mistrust of the medical system, stemming from a history of transgressions such as the Tuskegee syphilis trials and the theft of Henrietta Lacks’ cells. The confluence of these factors can make a timely diagnosis and adequate treatment exponentially harder, contributing to serious health consequences and resulting in care that falls flat.
Approximately 20% of drugs have different effects depending on a patient’s race, which makes racial representation in clinical trials essential to developing effective treatments. However, people of color are critically underrepresented in research. Estimates show that non-white people make up less than 16% of research study participants, despite representing more than 38% of the U.S. population.
In an effort to better align clinical trial enrollment to reflect real-world patient populations, the FDA issued draft guidance in April 2022 to help companies enroll clinical trial participants from marginalized racial and ethnic groups. This guidance was reinforced by Public Law 117-328 in December 2022, which now requires clinical trials to develop diversity action plans.
Although a significant step in the right direction, a critical missing element is penalties or incentives to ensure compliance. History has made it all too clear that widespread changes to support underrepresented populations are rarely made because they are the right thing to do but rather by positive incentivization or fear of repercussions.
Furthermore, we are now seeing companies recruiting people of color for clinical trials in a perfunctory attempt to simply “check the box” — and I say that is no better than tokenism. The message I have heard from communities of color is clear: Do not simply show up and ask for help so you can use marginalized people suffering from rare diseases for your own benefit. Patients of color with rare diseases are not racial tokens.
At its core, tokenism is the practice of including individuals from underrepresented groups to create an appearance of diversity to meet minimum requirements. This transactional approach undermines the meaningful inclusion of diverse populations by reducing their participation to a mere symbolic gesture. Because there is a long history of not including marginalized communities in healthcare research, the very fact that the FDA was compelled to require the development of diversity action plans, rather than the industry driving this change themselves, is a clear indicator of tokenism at play. In the wake of Public Law 117-328, the underlying motivation for recruiting vulnerable communities is undoubtedly being driven more by the need for compliance than a genuine desire to improve outcomes for diverse populations. Today’s clinical trial recruitment tactics are centered on meeting quotas and making pharmaceutical companies look good.
Tokenism is discrimination. It leaves people voiceless and feeling like they are being used. This transactional approach can undermine the trust of communities, decrease engagement and participation, and perpetuate existing inequalities. Including diverse populations in the research phase of clinical trials is undeniably a positive step, but it’s ultimately nothing more than tokenism if the audience being recruited isn’t involved in planning and decision-making. The FDA’s current guidelines don’t require input or representation of diverse communities in the development of diversity action plans. To move beyond tokenism and transactional motivations, it is crucial to embrace an authentic commitment to inclusivity and representation for better, more equitable healthcare.
To foster genuine engagement, clinical trial providers should prioritize building relationships with diverse communities, understanding their specific concerns and needs, and involving them in all stages of the research process. This includes collaborating with community leaders and organizations, conducting culturally sensitive recruitment and outreach efforts, providing necessary support and resources, and ensuring that trial design considers the diverse characteristics and health profiles of the participants.
I have personally experienced that when organizations establish trust and foster relationships with underrepresented communities, they experience increased willingness to engage. What we need from pharmaceutical companies is a genuine desire for inclusion and meaningful collaboration. Simply put, show that you really care.
The reality is that the U.S. demographics for race and ethnicity are expected to have more Black and brown people than white people in the next 20 or so years. As we look to the future, let us not forget that the health of our society is only as strong as the health of our most vulnerable communities. Let us strive to ensure that clinical trial recruitment practices are inclusive and representative so we can provide the best standard of care for everyone. After all, when the "minorities" become the majority, we want to be ready to meet their needs and ensure that all are supported.
About The Author:
Jenifer Ngo Waldrop joined the Black Women’s Health Imperative (BWHI) as executive director of the Rare Disease Diversity Coalition (RDDC) in October 2022. Before joining the leadership team of RDDC, she developed models and programs for diversity, equity, and inclusion initiatives to support multiple business units with Fortune 500 companies and organizations in Asia, Canada, and the U.S.