Engage Health, Inc. presented an abstract and poster entitled ‘Rare Disease Qualitative Research: Methodologies for Conceptual Strength Regarding the Burden of Access’ at the Virtual World Conference on Rare Diseases.

Capturing the experience of those navigating the United States healthcare system is challenging; each state has different resources, each program has different rules, and these combinations often result in different outcomes. Within the rare disease space, this process has even more variability based on where a patient lives, how knowledgeable they are, and their type of health insurance coverage. The authors attempted to address these issues by capturing the experience of families living with a specific rare disease through a novel methodology which focuses on capturing experiences through the voice of patients and families.

Using this methodology, the researchers were able to obtain meaningful information that was clear and representative of a small sample of patients/families impacted by a group of rare diseases and its subtypes. These findings will be utilized by the patient organization to design and offer programming to reduce gaps in understanding, strengthen health insurance literacy, and provide baseline data on community experiences as new treatment options are becoming available.