Comparison of Rare Disease Patient and Caregiver Perceptions Regarding Access to Disease Information and Interactions with their Healthcare Providers: Results from an International Survey

Author(s)

Siva Narayanan, MPhil, MS, MSc, PhD1, Wesley Michael, MBA2;
1Avant Health, CEO, Bethesda, MD, USA, 2Rare Patient Voice LLC, Towson, MD, USA
OBJECTIVES: To assess perceptions of rare disease (RD) patients and their caregivers regarding access to RD information and interactions with their healthcare providers (HCPs).
METHODS: Eight hundred participants were recruited for an international survey, leveraging a RD stakeholder panel. All participants had to be a RD patient or a caregiver of RD patient, or RD patient who was also a caregiver (“both”), >=18 years of age and reside in Germany, Spain, the United Kingdom (UK) or the United States (U.S). Participants completed an online survey outlining their (or their family member’s) experience living with RD, including information about interactions with their HCPs.
RESULTS: Survey participants included 100 from Germany, 168 from Spain, 252 from the UK and 280 from the U.S; 78% were RD patients, 15% were caregivers of a RD patient, and 7% represented “both” groups. Over 300 unique RDs were represented within the study cohort. Majority (72%/61%/75% of patients/caregivers/both) reported having difficulties attaining information regarding their RD or new treatment options under development for their condition; 48%/33%/48% of patients/caregivers/both reported that their medical team is never/rarely educated about their RD. Only 12%/22%/13% of patients/caregivers/both reported ‘often/always’ receiving education about their RD from their medical team and only 27%/33%/25% of patients/caregivers/both reported that the information received is ‘often/always’ easy to understand. Furthermore, only 32%/10%/34% of patients/caregivers/both reported “often/always” that their needs were heard by their medical team.
CONCLUSIONS: Majority of the RD patients and caregivers reported difficulties attaining information regarding their RD or new treatment options, and reported rarely receiving RD information from their medical team and that the received information was not easy to understand. Perceptions varied between patients and caregivers. Better strategies to improve knowledge and awareness of RD among HCPs, and better communication of RD information to patients/caregivers is warranted.

Conference/Value in Health Info

2025-05, ISPOR 2025, Montréal, Quebec, CA

Value in Health, Volume 28, Issue S1

Code

PCR147

Topic

Patient-Centered Research

Topic Subcategory

Patient Engagement, Patient-reported Outcomes & Quality of Life Outcomes

Disease

No Additional Disease & Conditions/Specialized Treatment Areas, SDC: Rare & Orphan Diseases

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