COMMONALITIES IN DISEASE BURDEN AND UNMET NEED IN UNDERSERVED AUTOIMMUNE DISEASES
Author(s)
Sebastian Vermeersch, MSc, PhD1, Lies Schoonaert, PhD2, Lutgarde Allard, -3, Coralie Bouillot, -4, Nancy Di Salvo, -5, Mervyn Morgan, -6, Jean-Philippe Plançon, -7, Yves Piette, MD8, Renee Campbell, MBA, MPH, MD9, deMauri Mackie, PhD10, Chris Leo Pashos, BS, MPP, PhD11, Glenn A. Phillips, PhD12, Julien Patris, MA13.
1Senior Manager, hict, Gent, Belgium, 2Hict, Gent, Belgium, 3EuMGA, La Louvière, Belgium, 4Sjögren Europe, Gland, Switzerland, 5GBS | CIDP Foundation International, Conshohocken, PA, USA, 6ITP Support Association (United Kingdom & Ireland), Bolnhurst, United Kingdom, 7EPODIN, Le Pouliguen, France, 8UZ Gent, Gent, Belgium, 9argenx, brooklyn, NY, USA, 10argenx, Elkins Park, PA, USA, 11argenx, Winchester, MA, USA, 12argenx, Boston, MA, USA, 13argenx, Gent, Belgium.
1Senior Manager, hict, Gent, Belgium, 2Hict, Gent, Belgium, 3EuMGA, La Louvière, Belgium, 4Sjögren Europe, Gland, Switzerland, 5GBS | CIDP Foundation International, Conshohocken, PA, USA, 6ITP Support Association (United Kingdom & Ireland), Bolnhurst, United Kingdom, 7EPODIN, Le Pouliguen, France, 8UZ Gent, Gent, Belgium, 9argenx, brooklyn, NY, USA, 10argenx, Elkins Park, PA, USA, 11argenx, Winchester, MA, USA, 12argenx, Boston, MA, USA, 13argenx, Gent, Belgium.
OBJECTIVES: We tested the feasibility of synthesizing evidence across multiple autoimmune conditions to (1) explore the concept of 'underserved autoimmunity' through shared dimensions of unmet need, (2) characterize the collective burden across those dimensions, and (3) surface evidence and awareness gaps that prevent recognition and action.
METHODS: We conducted an exploratory mapping of published and real-world evidence using the KCE NEED framework (2025), a European framework for assessing patients' and societal unmet health-related needs, as organizing structure. We initially focused on myasthenia gravis and chronic inflammatory demyelinating polyneuropathy, subsequently extending to immune thrombocytopenic purpura, Sjögren's disease, and multifocal motor neuropathy. Evidence came from a pragmatic literature review. Findings were validated through two European multi-stakeholder (physician and patient advocates) advisory boards in October 2025 and May 2026.
RESULTS: Evidence mapping identified cross-cutting dimensions of unmet need as a central theme of underserved autoimmune diseases: persistent diagnostic delays compounded by low clinical awareness, standard of care frequently relying on broad immunosuppressive approaches, and limited therapeutic innovation relative to disease burden. Participants validated the psychological toll, patient and caregiver burden across financial and social dimensions, and working-age productivity loss as key drivers of the burden profile. A patient-clinician gap in burden assessment was flagged as a structural barrier to disease recognition. Evidence was relatively well developed for patient health and healthcare use, but sparse or absent for societal burden, caregiver impact, and the burden carried by undiagnosed or misdiagnosed patients.
CONCLUSIONS: This proof-of-concept synthesis demonstrates that a recognizable burden profile emerges across underserved autoimmune conditions spanning different therapeutic areas, with shared dimensions of unmet need; additional research is required to further define them. These shared dimensions support substantial unmet need in underserved autoimmune diseases, and makes the case for systematic evidence generation to inform regulatory, access, and treatment decisions and awareness initiatives.
METHODS: We conducted an exploratory mapping of published and real-world evidence using the KCE NEED framework (2025), a European framework for assessing patients' and societal unmet health-related needs, as organizing structure. We initially focused on myasthenia gravis and chronic inflammatory demyelinating polyneuropathy, subsequently extending to immune thrombocytopenic purpura, Sjögren's disease, and multifocal motor neuropathy. Evidence came from a pragmatic literature review. Findings were validated through two European multi-stakeholder (physician and patient advocates) advisory boards in October 2025 and May 2026.
RESULTS: Evidence mapping identified cross-cutting dimensions of unmet need as a central theme of underserved autoimmune diseases: persistent diagnostic delays compounded by low clinical awareness, standard of care frequently relying on broad immunosuppressive approaches, and limited therapeutic innovation relative to disease burden. Participants validated the psychological toll, patient and caregiver burden across financial and social dimensions, and working-age productivity loss as key drivers of the burden profile. A patient-clinician gap in burden assessment was flagged as a structural barrier to disease recognition. Evidence was relatively well developed for patient health and healthcare use, but sparse or absent for societal burden, caregiver impact, and the burden carried by undiagnosed or misdiagnosed patients.
CONCLUSIONS: This proof-of-concept synthesis demonstrates that a recognizable burden profile emerges across underserved autoimmune conditions spanning different therapeutic areas, with shared dimensions of unmet need; additional research is required to further define them. These shared dimensions support substantial unmet need in underserved autoimmune diseases, and makes the case for systematic evidence generation to inform regulatory, access, and treatment decisions and awareness initiatives.
Conference/Value in Health Info
2026-11, ISPOR Europe 2026, Vienna, Austria
Value in Health, Volume 29, Issue 12S
Code
EPH149
Topic
Epidemiology & Public Health, Patient-Centered Research
Disease
Systemic Disorders/Conditions (Anesthesia, Auto-Immune Disorders (n.e.c.), Hematological Disorders (non-oncologic), Pain)