QUANTIFYING UNMET NEEDS IN RARE OR SEVERE AUTOIMMUNE DISEASES: NOVEL STUDY DESIGN FOR A CROSS-INDICATION COMPARISON OF PATIENT AND PHYSICIAN PREFERENCES
Author(s)
Renee Campbell, MBA, MPH, MD1, Julien Patris, MA2, Glenn A. Phillips, PhD3, deMauri Mackie, PhD4, Adam Moore, BSc, PhD5, Laurie Batchelder, BA, MSc, PhD6, Erica Visintin, PhD7.
1argenx, brooklyn, NY, USA, 2argenx, ghent, Belgium, 3Argenx, Boston, MA, USA, 4argenx, Elkins Park, PA, USA, 5IQVIA, London, United Kingdom, 6IQVIA, Reading, United Kingdom, 7IQVIA Ltd, London, United Kingdom.
1argenx, brooklyn, NY, USA, 2argenx, ghent, Belgium, 3Argenx, Boston, MA, USA, 4argenx, Elkins Park, PA, USA, 5IQVIA, London, United Kingdom, 6IQVIA, Reading, United Kingdom, 7IQVIA Ltd, London, United Kingdom.
OBJECTIVES: Rare or severe autoimmune diseases are heterogeneous, yet patients often experience common, recurring burdens. Symptom burden is multi-dimensional and frequently invisible; fatigue, pain, and cognitive symptoms can substantially impact daily life, including financial, productivity, and caregiver outcomes. Patient and physician perceptions of unmet needs may diverge when symptoms are invisible or persist over time, or when precision therapies are lacking. This study aims to generate evidence of unmet needs across rare or severe autoimmune diseases, including myasthenia gravis, chronic inflammatory demyelinating polyradiculoneuropathy, multifocal motor neuropathy, myositis, and primary Sjögren’s disease, identifying similarities and differences across conditions and between patients and healthcare providers (HCPs).
METHODS: A staged, mixed-methods, international preference study aligned with the Innovative Medicines Initiative Patient Preferences in Benefit-Risk Assessments framework will be conducted in four stages: (1) evidence mapping to identify unmet-need concepts and map statements to Needs Examination, Evaluation, and Dissemination (NEED) criteria; (2) qualitative interviews with patients and HCPs to prioritise attributes and inform non-preference survey questions; (3) a steering committee workshop to validate attributes and finalise preference method; and (4) a survey to quantify unmet needs across selected NEED dimensions.
RESULTS: This approach will generate a traceable attribute set mapped to selected NEED dimensions, a survey instrument validated by patients and HCPs, and relative importance weights and unmet-need gap estimates supporting patient-clinician and cross-indication comparisons. The integrated framework will provide a rigorous, reproducible method for quantifying unmet needs beyond traditional clinical measures and assessing consistency across conditions.
CONCLUSIONS: This study will provide an approach for identifying commonalities and differences in unmet needs across rare or severe autoimmune conditions and among patients and HCPs. Evidence of consistent unmet-need gaps in these conditions may have significant implications for future clinical research, as well as for adjusting regulatory and access frameworks and clinical practice.
METHODS: A staged, mixed-methods, international preference study aligned with the Innovative Medicines Initiative Patient Preferences in Benefit-Risk Assessments framework will be conducted in four stages: (1) evidence mapping to identify unmet-need concepts and map statements to Needs Examination, Evaluation, and Dissemination (NEED) criteria; (2) qualitative interviews with patients and HCPs to prioritise attributes and inform non-preference survey questions; (3) a steering committee workshop to validate attributes and finalise preference method; and (4) a survey to quantify unmet needs across selected NEED dimensions.
RESULTS: This approach will generate a traceable attribute set mapped to selected NEED dimensions, a survey instrument validated by patients and HCPs, and relative importance weights and unmet-need gap estimates supporting patient-clinician and cross-indication comparisons. The integrated framework will provide a rigorous, reproducible method for quantifying unmet needs beyond traditional clinical measures and assessing consistency across conditions.
CONCLUSIONS: This study will provide an approach for identifying commonalities and differences in unmet needs across rare or severe autoimmune conditions and among patients and HCPs. Evidence of consistent unmet-need gaps in these conditions may have significant implications for future clinical research, as well as for adjusting regulatory and access frameworks and clinical practice.
Conference/Value in Health Info
2026-11, ISPOR Europe 2026, Vienna, Austria
Value in Health, Volume 29, Issue 12S
Code
MSR208
Topic
Methodological & Statistical Research
Topic Subcategory
Survey Methods
Disease
Rare & Orphan Diseases, Systemic Disorders/Conditions (Anesthesia, Auto-Immune Disorders (n.e.c.), Hematological Disorders (non-oncologic), Pain)