(261) Outcome Measures Relevant for Assessing Health Related Quality of Life and Utility in Friedreich Ataxia

Friday, August 26, 2022

8:00 AM – 6:00 PM CEST

Location: Hall C

Publication Number: 592

Presenting Author(s)

Christina J. Qian, MSc
– Associate Director, Broadstreet HEOR, Vancouver, British Columbia, Canada

Co-Author(s)

Katharina Buesch
– PTC Therapeuticsw, Steinhausen/Zug, Zug, Switzerland
Tina Li, MPH
– Research Scientist, Broadstreet HEOR, Canada
Andrea Bever, MPH
– Research Scientist, Broadstreet HEOR, BC, Canada
Jessica Dunne, BSc (OT), MSc
– Research Scientist, Broadstreet Health Economics & Outcomes Research, Vancouver, BC, Canada
Shelagh Szabo, MSc
– CEO and Scientific Director, Broadstreet HEOR, Canada

Background: The progression of Friedreich Ataxia (FA) can result in severe disability, dramatically affecting the health-related quality of life (HRQoL) of patients and caregivers. New therapies are under development and will require utility estimates for health states characterizing the HRQoL impact of FA.

Objectives: This study aimed to compare the coverage of domains, validity, feasibility, and acceptability of generic preference-based measures (PBMs) for assessing the impact of FA on the HRQoL of patients and caregivers.

Methods: Targeted literature reviews were conducted to identify drivers of HRQoL among FA patients and their caregivers, and relevant outcome measures to capture these impacts. Publications describing these measures were reviewed to understand coverage of relevant HRQoL drivers, validity for FA, respondent burden, and acceptability to selected health technology assessment agencies.

Results: Key drivers of HRQoL among both patients and caregivers included impacts on fatigue/sleep, emotion/mood, and social interactions. Additional drivers were impacts on activities of daily living and physical function for patients; and impact on productivity and leisure activities, pain, and physical health for caregivers. Relevant generic PBMs identified during the review included the EQ-5D, Health Utilities Index (HUI), SF-6D, Quality of Well-Being Scale (QWB), Child Health Utility 9D (CHU9D), Assessment of Quality of Life (AQoL), Care-related Quality of Life (CarerQoL), Carer Experience Scale (CES), and Adult Social Care Outcomes Toolkit for Carers (ASCOT-Carer). While most directly or indirectly covered many of the drivers, the SF-6D, EQ-5D, and HUI2 had the strongest balance of driver coverage, validity, feasibility, and acceptability among patient outcome measures; and the EQ-5D, CarerQoL, and ASCOT-Carer among caregiver outcome measures.

Conclusions: Generic PBMs covering FA-relevant constructs can provide important insights into the burden experienced by patients and their caregivers. These findings will help inform the choice of outcome measures for HRQoL studies in FA and upcoming health economic analyses.