From Pain to Patient Preferences Patient Engagement in Early HTA for Repurposing Losartan in Osteogenesis Imperfecta
Author(s)
Dalma Hosszú, MA1, Eve Hewitt, BSc2, Zsuzsa Reka Pozsar, MA3, Claudia Fuchs, PhD4, Judith Cohen, PhD5, Nick J. Bishop, PhD6, Donald Lo, PhD7, Zoltan Kalo, PhD8, Antal Tamas Zemplenyi, MSc, PhD9.
1Institute of Psychology, University of Pécs & Syreon Research Institute, Budapest, Hungary, 2Beacon for Rare Diseases, Cambridge, United Kingdom, 3Center for Health Technology Assessment, Semmelweis University; Center for Pharmacology and Drug Research & Development, Semmelweis University & Syreon Research Institute, Budapest, Hungary, 4EURORDIS - Rare Disease Europe, Brussels, Belgium, 5University of Hull, Hull, United Kingdom, 6University of Sheffield, Sheffield, United Kingdom, 7European Infrastructure for Translational Medicine (EATRIS), Amsterdam, Netherlands, 8Center for Health Technology Assessment, Semmelweis University & Syreon Research Institute, Budapest, Hungary, 9Faculty of Pharmacy Center for Health Technology Assessment and Pharmacoeconomic Research, University of Pécs, Hungary & University of Colorado Skaggs School of Pharmacy and Pharmaceutical Science, Denver, CO, USA.
1Institute of Psychology, University of Pécs & Syreon Research Institute, Budapest, Hungary, 2Beacon for Rare Diseases, Cambridge, United Kingdom, 3Center for Health Technology Assessment, Semmelweis University; Center for Pharmacology and Drug Research & Development, Semmelweis University & Syreon Research Institute, Budapest, Hungary, 4EURORDIS - Rare Disease Europe, Brussels, Belgium, 5University of Hull, Hull, United Kingdom, 6University of Sheffield, Sheffield, United Kingdom, 7European Infrastructure for Translational Medicine (EATRIS), Amsterdam, Netherlands, 8Center for Health Technology Assessment, Semmelweis University & Syreon Research Institute, Budapest, Hungary, 9Faculty of Pharmacy Center for Health Technology Assessment and Pharmacoeconomic Research, University of Pécs, Hungary & University of Colorado Skaggs School of Pharmacy and Pharmaceutical Science, Denver, CO, USA.
OBJECTIVES: Patient engagement is essential of aligning drug development with unmet needs and meaningful outcomes, supporting access decisions and early identification of non-viable projects. REMEDi4ALL is an EU-funded initiative for drug repurposing, embedding early HTA and patient engagement to guide losartan’s development in osteogenesis imperfecta (OI). While fracture reduction is a key clinical outcome in trials and is targeted with treatments, patients indicated that their unmet needs were not fully represented by this outcome. This work aimed to define a disease-specific, patient-informed unmet need list for OI.
METHODS: A multi-stakeholder workshop in 2024 explored patient preferences in OI using an initial element list. This session laid the foundation for a June 2025 workshop at the OIFE annual meeting with 31 patients. After presenting examples of unmet need from OI literature, participants expressed their specific unmet needs in facilitated groups. Subsequently written outputs were synthesised into domains by HEOR researchers.
RESULTS: Twenty distinct unmet need elements were identified and grouped into three domains: Therapeutic Gaps (6), Clinical Care Gaps (9), and Supportive Care Gaps (5). These domains cover a broad range of patient-centric value drivers that go beyond conventional clinical endpoints. Chronic pain, fatigue, and mental health challenges were consistently mentioned across workshops, reflecting their impact on daily functioning and overall quality of life.
CONCLUSIONS: The identified unmet needs directly informed how treatment benefits were communicated in patient-support materials, including revisions to the patient information leaflet to reflect priorities such as pain and fatigue. This input contributed to a shift in the target product profile: while fracture reduction remained the central target for drug action, greater emphasis was placed on pain and fatigue reduction being leading therapeutic benefits. Incorporating patient perspectives early provided insights into enrolment barriers and communication needs, improved evidence generation strategies, and will support better alignment between value propositions and patient priorities.
METHODS: A multi-stakeholder workshop in 2024 explored patient preferences in OI using an initial element list. This session laid the foundation for a June 2025 workshop at the OIFE annual meeting with 31 patients. After presenting examples of unmet need from OI literature, participants expressed their specific unmet needs in facilitated groups. Subsequently written outputs were synthesised into domains by HEOR researchers.
RESULTS: Twenty distinct unmet need elements were identified and grouped into three domains: Therapeutic Gaps (6), Clinical Care Gaps (9), and Supportive Care Gaps (5). These domains cover a broad range of patient-centric value drivers that go beyond conventional clinical endpoints. Chronic pain, fatigue, and mental health challenges were consistently mentioned across workshops, reflecting their impact on daily functioning and overall quality of life.
CONCLUSIONS: The identified unmet needs directly informed how treatment benefits were communicated in patient-support materials, including revisions to the patient information leaflet to reflect priorities such as pain and fatigue. This input contributed to a shift in the target product profile: while fracture reduction remained the central target for drug action, greater emphasis was placed on pain and fatigue reduction being leading therapeutic benefits. Incorporating patient perspectives early provided insights into enrolment barriers and communication needs, improved evidence generation strategies, and will support better alignment between value propositions and patient priorities.
Conference/Value in Health Info
2025-11, ISPOR Europe 2025, Glasgow, Scotland
Value in Health, Volume 28, Issue S2
Code
PCR100
Topic
Patient-Centered Research
Topic Subcategory
Patient Engagement, Patient-reported Outcomes & Quality of Life Outcomes
Disease
Mental Health (including addition), Rare & Orphan Diseases