APTITUDE TOWARDS PATIENT-CENTERED CARE AMONG PHYSICIANS MANAGING RARE DISEASES IN THE UNITED STATES: A CROSS-SECTIONAL SURVEY
Author(s)
Alissar Moussallem, PharmD, MPH1, Tim Irfan, MBA2, Stacey Purinton, MBA, MPH, RN3, Perrine Le Calvé, MSc1, Amina Omri, MA1, TAREK MNIF, PharmD1, Vicky Nogueira Pileggi, PhD4, Bastien Vincent, MSc1, Franco Esposito, BSc5, Tanya Louise Collin-Histed, .6, Suzanne Reed, BA, MSc, PhD1.
1Oracle Life Sciences, Paris, France, 2Oracle Life Sciences, Munich, Germany, 3Oracle Life Sciences, Kansas City, MO, USA, 4Oracle Life Sciences, São Paulo, Brazil, 5All Global, London, United Kingdom, 6International Gaucher Alliance, London, United Kingdom.
1Oracle Life Sciences, Paris, France, 2Oracle Life Sciences, Munich, Germany, 3Oracle Life Sciences, Kansas City, MO, USA, 4Oracle Life Sciences, São Paulo, Brazil, 5All Global, London, United Kingdom, 6International Gaucher Alliance, London, United Kingdom.
OBJECTIVES: To describe patient-centered practices, including communication, empathy, and involvement of patients and caregivers in decision-making among physicians managing patients with rare diseases.
METHODS: This was a cross-sectional study conducted among primary care physicians (PCPs) and specialists in the United States (US), recruited via physician panels. Data were collected using a structured questionnaire between October and November 2025.
RESULTS: A total of 617 physicians participated in the study, including 206 PCPs and 411 specialists; 80.0% of them had at least 10 years of clinical experience. Most physicians (88.7%) agreed that it is important to encourage patients and caregivers’ involvement in decision-making. Physicians reported a high degree of involvement in treatment initiation or switching discussions with patients and caregivers. The majority reported a large extent of patient involvement in decision-making (79.3%), including active participation (74.6%), physicians’ consideration of patient’s perspective (72.8%), and mutual exchange of information (68.9%). Around a third of physicians (186/617) agreed and strongly agreed that patients and/or caregivers are experts in their rare disease. Many reported learning disease-related information from patients, including general symptomology (73.9%), burden of the disease (71.5%), natural history of the disease (70.0%), and available treatment options (68.7%). PCPs were more likely than specialists to learn about available treatments (76.7% vs 64.7%, p=0.002) and disease burden (76.7% vs 68.9%, p=0.042). More than 90% of physicians agreed that empathy can build trust and enhances their relationship with their patients, a similar attitude was observed between PCPs and specialists. Discussions around quality of life were commonly reported across the various disease stages, most frequently throughout the treatment journey (70.7%).
CONCLUSIONS: Physicians managing rare diseases in the US report high levels of patient and caregiver involvement in clinical decision-making and acknowledge the lived experiences of patients. These findings underscore the importance of empathetic, collaborative approaches to support patient-centered care in rare diseases.
METHODS: This was a cross-sectional study conducted among primary care physicians (PCPs) and specialists in the United States (US), recruited via physician panels. Data were collected using a structured questionnaire between October and November 2025.
RESULTS: A total of 617 physicians participated in the study, including 206 PCPs and 411 specialists; 80.0% of them had at least 10 years of clinical experience. Most physicians (88.7%) agreed that it is important to encourage patients and caregivers’ involvement in decision-making. Physicians reported a high degree of involvement in treatment initiation or switching discussions with patients and caregivers. The majority reported a large extent of patient involvement in decision-making (79.3%), including active participation (74.6%), physicians’ consideration of patient’s perspective (72.8%), and mutual exchange of information (68.9%). Around a third of physicians (186/617) agreed and strongly agreed that patients and/or caregivers are experts in their rare disease. Many reported learning disease-related information from patients, including general symptomology (73.9%), burden of the disease (71.5%), natural history of the disease (70.0%), and available treatment options (68.7%). PCPs were more likely than specialists to learn about available treatments (76.7% vs 64.7%, p=0.002) and disease burden (76.7% vs 68.9%, p=0.042). More than 90% of physicians agreed that empathy can build trust and enhances their relationship with their patients, a similar attitude was observed between PCPs and specialists. Discussions around quality of life were commonly reported across the various disease stages, most frequently throughout the treatment journey (70.7%).
CONCLUSIONS: Physicians managing rare diseases in the US report high levels of patient and caregiver involvement in clinical decision-making and acknowledge the lived experiences of patients. These findings underscore the importance of empathetic, collaborative approaches to support patient-centered care in rare diseases.
Conference/Value in Health Info
2026-05, ISPOR 2026, Philadelphia, PA, USA
Value in Health, Volume 29, Issue S6
Code
HSD66
Topic
Health Service Delivery & Process of Care
Disease
SDC: Rare & Orphan Diseases