APPLYING THE KCE NEED FRAMEWORK TO CHARACTERIZE BURDEN AND UNMET NEED ACROSS UNDERSERVED AUTOIMMUNE DISEASES: A MULTI-STAKEHOLDER METHODS STUDY
Author(s)
Sebastian Vermeersch, MSc, PhD1, Lies Schoonaert, PhD2, Coralie Bouillot, -3, Nancy Di Salvo, -4, Mervyn Morgan, -5, Jean-Philippe Plançon, -6, Yves Piette, MD7, Renee Campbell, MBA, MPH, MD8, deMauri Mackie, PhD9, Chris Leo Pashos, BS, MPP, PhD10, Glenn A. Phillips, PhD11, Julien Patris, MA12.
1Senior Manager, hict, Gent, Belgium, 2Hict, Ghent, Belgium, 3Sjögren Europe, Gland, Switzerland, 4GBS | CIDP Foundation International, Conshohocken, PA, USA, 5ITP Support Association (United Kingdom & Ireland), Bolnhurst, United Kingdom, 6EPODIN, Le Pouliguen, France, 7UZ Gent, Gent, Belgium, 8argenx, brooklyn, NY, USA, 9argenx, Elkins Park, PA, USA, 10argenx, Winchester, MA, USA, 11argenx, Boston, MA, USA, 12argenx, Gent, Belgium.
1Senior Manager, hict, Gent, Belgium, 2Hict, Ghent, Belgium, 3Sjögren Europe, Gland, Switzerland, 4GBS | CIDP Foundation International, Conshohocken, PA, USA, 5ITP Support Association (United Kingdom & Ireland), Bolnhurst, United Kingdom, 6EPODIN, Le Pouliguen, France, 7UZ Gent, Gent, Belgium, 8argenx, brooklyn, NY, USA, 9argenx, Elkins Park, PA, USA, 10argenx, Winchester, MA, USA, 11argenx, Boston, MA, USA, 12argenx, Gent, Belgium.
OBJECTIVES: We explored how KCE NEED (2025), a European framework for assessing patients' and societal unmet health-related needs, maps onto underserved autoimmune diseases as a cross-specialty category, aiming to (1) assess cross-specialty applicability, (2) identify which dimensions most consistently capture the burden profile of this disease group, and (3) characterize where dimension priorities converge and diverge across physician and patient advocate perspectives.
METHODS: We applied the KCE NEED framework to evidence initially anchored in myasthenia gravis and chronic inflammatory demyelinating polyneuropathy and extended to conditions across neurology, hematology, and rheumatology. Framework fit was assessed over two European multi-stakeholder advisory boards (October 2025; May 2026) involving 9 physicians and 7 patient advocates from 7 countries. The first board validated burden dimensions against real-world experience; the second examined NEED dimensions and cross-specialty applicability.
RESULTS: Advisory board participants confirmed a three-domain structure as best capturing the burden profile of underserved autoimmune diseases, with equity transversal. In the health domain, impact on physical and psychological health, HRQoL, and autonomy were confirmed across specialties and stakeholders. In the healthcare domain, quality of care, accessibility, burden of treatment, standard of care limitations, and impact of healthcare system pressures on care delivery were confirmed across specialties and stakeholders. In the social domain, impact on education, work, and social life and burden on informal caregivers were confirmed but showed the greatest divergence between patient and clinician perspectives. Patient advocates consistently flagged fatigue, cognitive difficulties, and social isolation as underweighted in clinical assessment.
CONCLUSIONS: Applying the NEED framework through a structured multi-stakeholder process yields a three-domain burden structure - health, healthcare, and social, with equity transversal - as a useful framework for characterizing underserved autoimmune diseases. Convergence across stakeholders was strongest in the health and healthcare domains; the social domain revealed the most meaningful divergence between patient and clinician perspectives.
METHODS: We applied the KCE NEED framework to evidence initially anchored in myasthenia gravis and chronic inflammatory demyelinating polyneuropathy and extended to conditions across neurology, hematology, and rheumatology. Framework fit was assessed over two European multi-stakeholder advisory boards (October 2025; May 2026) involving 9 physicians and 7 patient advocates from 7 countries. The first board validated burden dimensions against real-world experience; the second examined NEED dimensions and cross-specialty applicability.
RESULTS: Advisory board participants confirmed a three-domain structure as best capturing the burden profile of underserved autoimmune diseases, with equity transversal. In the health domain, impact on physical and psychological health, HRQoL, and autonomy were confirmed across specialties and stakeholders. In the healthcare domain, quality of care, accessibility, burden of treatment, standard of care limitations, and impact of healthcare system pressures on care delivery were confirmed across specialties and stakeholders. In the social domain, impact on education, work, and social life and burden on informal caregivers were confirmed but showed the greatest divergence between patient and clinician perspectives. Patient advocates consistently flagged fatigue, cognitive difficulties, and social isolation as underweighted in clinical assessment.
CONCLUSIONS: Applying the NEED framework through a structured multi-stakeholder process yields a three-domain burden structure - health, healthcare, and social, with equity transversal - as a useful framework for characterizing underserved autoimmune diseases. Convergence across stakeholders was strongest in the health and healthcare domains; the social domain revealed the most meaningful divergence between patient and clinician perspectives.
Conference/Value in Health Info
2026-11, ISPOR Europe 2026, Vienna, Austria
Value in Health, Volume 29, Issue 12S
Code
HTA202
Topic
Health Technology Assessment
Topic Subcategory
Value Frameworks & Dossier Format
Disease
Systemic Disorders/Conditions (Anesthesia, Auto-Immune Disorders (n.e.c.), Hematological Disorders (non-oncologic), Pain)