COMPARING PATIENT PERSPECTIVES AND HTA REQUIREMENTS IN HEALTH ECONOMIC MODELING: A MULTIPLE SCLEROSIS PERSPECTIVE
Author(s)
Jet Neervoort, Msc, Evie Groenewegen, MSc, Amber Tiemens, MSc, Sara W. Quist, MSc.
Asc Academics, Groningen, Netherlands.
Asc Academics, Groningen, Netherlands.
OBJECTIVES: Given the increasing focus on the incorporation of patient perspectives into health economics and outcomes research, this study examines the extent to which cost-effectiveness models (CEMs) reflect both health technology assessment (HTA) requirements and patient needs.
METHODS: To explore how patient perspectives are captured in HTA, multiple sclerosis (MS) served as a case study. Six semi-structured interviews were conducted with three HTA experts (two from Zorginstituut Nederland [ZIN] and one from National Institute for Health and Care Excellence [NICE]), two MS clinicians, and one MS patient representative. Findings were analysed across four domains: patient-relevant outcomes, patient experience data, patient heterogeneity, and patient engagement.
RESULTS: The interviews revealed growing interest in incorporating patient perspectives into health economic modelling, reflecting a shared goal among patients and HTA bodies to improve health outcomes through clinically meaningful models. HTA bodies emphasise methodological standardisation and cross-technology comparability, relying on model-ready evidence. In contrast, MS patients prioritise outcomes reflecting daily life—fatigue, cognition, functioning, side effects, and treatment preferences—which are rarely captured in standard evidence packages. For example, in MS, fatigue and cognitive decline substantially affect quality of life, yet these effects are insufficiently captured by the EQ-5D. Because cross-technology comparisons rely on a common measure and the EQ-5D remains the accepted standard, patient-relevant outcomes may be overlooked in current CEMs. While disease-specific outcomes can provide additional context, they are generally considered supportive. Patient preferences and qualitative experiences face a similar challenge: recognised as relevant by some HTA bodies, but without formalised pathways into model parameters, their role remains largely contextual.
CONCLUSIONS: Patient-centred evidence can enhance the relevance of CEMs by capturing patient-relevant outcomes. However, its integration comes with barriers: concerns around subjectivity and bias, alongside limited methodological guidance. Progress requires collaboration between patients and HTA bodies to strengthen modelling literacy and develop robust approaches.
METHODS: To explore how patient perspectives are captured in HTA, multiple sclerosis (MS) served as a case study. Six semi-structured interviews were conducted with three HTA experts (two from Zorginstituut Nederland [ZIN] and one from National Institute for Health and Care Excellence [NICE]), two MS clinicians, and one MS patient representative. Findings were analysed across four domains: patient-relevant outcomes, patient experience data, patient heterogeneity, and patient engagement.
RESULTS: The interviews revealed growing interest in incorporating patient perspectives into health economic modelling, reflecting a shared goal among patients and HTA bodies to improve health outcomes through clinically meaningful models. HTA bodies emphasise methodological standardisation and cross-technology comparability, relying on model-ready evidence. In contrast, MS patients prioritise outcomes reflecting daily life—fatigue, cognition, functioning, side effects, and treatment preferences—which are rarely captured in standard evidence packages. For example, in MS, fatigue and cognitive decline substantially affect quality of life, yet these effects are insufficiently captured by the EQ-5D. Because cross-technology comparisons rely on a common measure and the EQ-5D remains the accepted standard, patient-relevant outcomes may be overlooked in current CEMs. While disease-specific outcomes can provide additional context, they are generally considered supportive. Patient preferences and qualitative experiences face a similar challenge: recognised as relevant by some HTA bodies, but without formalised pathways into model parameters, their role remains largely contextual.
CONCLUSIONS: Patient-centred evidence can enhance the relevance of CEMs by capturing patient-relevant outcomes. However, its integration comes with barriers: concerns around subjectivity and bias, alongside limited methodological guidance. Progress requires collaboration between patients and HTA bodies to strengthen modelling literacy and develop robust approaches.
Conference/Value in Health Info
2026-11, ISPOR Europe 2026, Vienna, Austria
Value in Health, Volume 29, Issue 12S
Code
PCR175
Topic
Economic Evaluation, Patient-Centered Research
Topic Subcategory
Patient Engagement, Patient-reported Outcomes & Quality of Life Outcomes
Disease
Neurological Disorders