Having one of the 7,000+ identified rare diseases can feel like a solitary struggle. But a pioneering study from the U.S. National Organization for Rare Disorders (NORD) aims to unite the voices of thousands of patients to demonstrate how rare conditions are experienced in the real world.
“Rare disease is a public health issue, but we lack the data to prove it,” NORD says on the research website. “By participating in the study and sharing your information, you'll help us show the significant unmet needs of the community, find solutions and advocate for greater support.”
According to NORD, one in 10 Americans lives with a rare medical condition, yet the data needed to understand the rare disease experience is lacking. NORD hopes the data collected will transform personal experiences into robust statistics, compelling policymakers and stakeholders to act and create programs that improve the lives of patients and caregivers.
So far, more than 2,000 people have volunteered to share their experiences. The goal is to follow thousands of U.S. participants over time and gather essential information related to health care issues, diagnosis, care and quality of life.
The National Organization for Rare Disorders, which advocates for patients with rare diseases, invites both patients and caregivers to register and log in to the IAMRARE® platform to share their experiences. The survey, available in English and Spanish, takes about an hour to complete.
Learn more about the Living Rare Study.
“Types of questions you might see are: what are the societal, health insurance, or system-imposed barriers to accessing diagnostic testing, care and treatment? How do these barriers differ across states, rare diseases and insurance types?” said Janine Lewis, Director of Research Operations at NORD.
To participate, patients complete a survey and return annually to update their responses if anything has changed. Participants will receive updates on study insights.
“By participating in this study, rare disease patients and caregivers have the opportunity to help others understand how to meet the needs of their community,” said Julieta Bonvin Sallago, Lead Clinical Research Associate at Connecticut Children’s and Assistant Professor at the University of Connecticut.
