What Rare Disease Patient Advocacy Groups Are Doing to Mitigate the Effects of Disparities

Abstract

Compared with chronic diseases affecting larger populations, rare disease (RD) patients experience great inequities in diagnosis, care, and research. Within RDs, health disparities compound these inequities, as marginalized communities experience additional barriers in accessing clinical care and are often underrepresented from participation in research and clinical trials. For almost 40 years, the National Organization for Rare Disorders (NORD), a RD umbrella organization with over 300 nonprofit organizational members, has led efforts to understand and address inequities for the RD community through innovative research, programming, and collaboration with patients, caregivers, practitioners, and external stakeholders. The beginning of the COVID-19 pandemic in 2020 brought to light longstanding disparities and discrimination for marginalized communities as well as pivotal racial justice movements. These events spurred many RD nonprofit organizations’ interest in increasing outreach and engagement with minoritized communities within RDs and diversifying their organization internally. Building on the increased interest in diversity, equity, and inclusion (DEI), NORD has focused on collecting case studies from within NORD and its member organizations to capture current efforts to improve DEI within the RD ecosystem. One way clinicians can work to mitigate the effects of disparities is to collaborate with RD patient organizations; this article provides a means by which clinicians and researchers can understand some of the challenges RD nonprofit organizations face in bridging disparities and learn about solutions to supporting marginalized patients within their communities. Clinicians are encouraged to join NORD in our policy efforts advocating to ensure patient access to health care providers practicing in a different state vis-à-vis telehealth.

Comparative policy analysis of national rare disease funding policies in Australia, Singapore, South Korea, the United Kingdom and the United States: a scoping review

“…Also worth mention is the numerous non-profit organizations in the US that provide support for rare disease research and advocate for patients [ 84 ]. These organizations often fundraise to support research, increase awareness, and assist patients with accessing and affording treatments.…”