We Need Equity and Diversity Within Rare Disease Research

Rare diseases face many obstacles when it comes to research and drug development. Small patient populations, a lack of awareness, and limited funding are just a few examples. When it comes to rare disease patients of color, there are even more barriers. Because people of color are often underrepresented in clinical trials, there is a poor understanding of how rare diseases impact them. Considering that a number of rare diseases – with sickle cell anemia acting as an example – are more likely to impact people of color, this lack of understanding is a major issue. There must be work done to increase equity and diversity within the rare disease landscape.

Challenges In Rare Disease Research

30 million people throughout the United States are impacted by a rare disease, which increases to 400 million when looking at the entire world. Half of these people are children. Despite the high number of patients, 95% of rare diseases do not have one treatment that has been approved by the FDA. Rare diseases are already poorly understood and researched, and rare diseases and their effects on people of color are even less understood and researched.

The answer to achieving a higher understanding is research, with clinical trials playing a large role. Fortunately, clinical trials have been adapting as the world of healthcare does, with the COVID-19 pandemic acting as the perfect example. Decentralized trials (DCTs) are now being used more heavily, allowing for greater access and the chance to reach more diverse patient populations.

While there have been improvements in access to clinical trials, the same problems that have always existed within the rare disease landscape still exist. These include small population sizes, which makes it difficult to find enough trial participants. Even if researchers are able to find a suitable number of people, they often come from all over the country or even the world. It creates an endless cycle that makes any research that much harder.

Luckily, medical professionals are working to address the problems. For one, there have been improvements in diagnosing rare diseases. New tools make diagnosis easier and faster, therefore increasing the sizes of patient populations. In addition, many pharmaceutical and biotechnology companies are adding departments that focus on rare diseases or dedicating more time and resources to rare disease research. Incentives from the FDA and other regulatory agencies have spurred this change, as they provide things like tax credits or priority review vouchers to companies developing treatments for rare conditions.

Equity, Diversity, and Rare Disease

The pandemic has shined a light on inequity and racism within the healthcare system. Access to healthcare is restricted for people of color and has been for generations. There must be more effort to increase diversity and equity within healthcare, research, and drug development.

In order to address this problem, sponsors should engage with patient communities that in authentic ways prove their long-term commitment. They should shift to see these communities as partners and ultimate stakeholders. In addition, there must be increased diversity when it comes to age, race gender, ethnicity, and sexual orientation. Policies should be implemented to ensure this occurs; they should mandate certain enrollment targets.

Organizations currently pushing for increased diversity and equity within the healthcare sphere include:

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