Presentation (CTI)

OBJECTIVES: Progressive supranuclear palsy (PSP) is a rare neurodegenerative disorder characterized by motor, cognitive, and ocular impairments, as well as speech and swallowing difficulties. There is currently no cure for PSP, with treatment options focusing on symptom management and quality of life improvement. Cost-utility analyses for PSP treatments require health state utilities, typically derived from generic preference-based measures (PBMs) such as EQ-5D, SF-6D, and Health Utilities Index (HUI2/3). However, the domains covered by these instruments may not adequately capture the full spectrum of PSP symptoms and impact. To evaluate generic preference-based measure relevance (EQ-5D, SF-6D, and HUI2/3) for generating health state utilities for PSP by mapping their domains to key disease features identified in the literature.
METHODS: A targeted literature review identified key PSP features, including gait/postural instability, speech/ocular dysfunction, cognitive impairment, emotional symptoms, swallowing difficulties, and urinary issues. Symptom extraction was performed by one reviewer and validated through discussion with a second reviewer. Concept mapping was conducted by two study team members to assess whether these features were directly or indirectly captured by each instrument’s dimensions.
RESULTS: EQ-5D partially assesses mobility and anxiety/depression but omits speech, ocular function, cognition, and swallowing. SF-6D captures mental health but lacks coverage of motor and ocular symptoms. HUI2 shows limited relevance with its cognition item focusing on school-related abilities; speech/vision are only partially addressed. HUI3 demonstrates the greatest overlap, directly assessing speech and cognition and partially capturing ambulation and vision. However, it still omits swallowing, urinary symptoms, and some movement features. Overall, all instruments exhibit substantial gaps in domain coverage for PSP.
CONCLUSIONS: Generic PBMs commonly recommended for health technology assessment (HTA) may be inappropriate for PSP due to limited content validity. Alternative approaches, such as vignette-based valuation, should be considered for utility estimation in PSP to ensure appropriate HTA of treatments for this rare condition.