Author
Listed:
- Maria Farris
(Novartis Pharmaceuticals Australia)
- Stephen Goodall
(University of Technology Sydney)
- Richard Abreu Lourenco
(University of Technology Sydney)
- Brendan Mulhern
(University of Technology Sydney)
- Kathleen Manipis
(University of Technology Sydney)
- Elena Meshcheriakova
(University of Technology Sydney)
- Milena Lewandowska
(University of Technology Sydney)
Abstract
Purpose Inherited retinal disease (IRD) causes progressive loss of visual function, degenerating towards complete blindness. Economic evaluation of gene therapies for rare forms of genetic IRDs have had to rely on health-related quality of life (HR-QoL) estimates from other diseases because there is limited data available for such a rare condition. This study aimed to estimate Australian societal-based utility values for IRD health states that can be used in cost-utility analyses (CUA) using a time trade-off (TTO) protocol adapted from a UK study. Methods The EuroQol Valuation Technology (EQVT) protocol composite TTO (cTTO) framework was followed, which includes worse-than-death (WTD) states and quality control (QC) measures. Preferences were collected from a general population sample of 110 Australian adult participants. Five health state vignettes from the UK study which had been validated with patients and clinicians were presented randomly to participants during videoconferencing (VC) interviews with one of four interviewers. Technical and protocol feasibility were assessed in a pilot of 10 interviews. QC measures were used to monitor interviewers’ performance during the study. Results One participant withdrew consent. The final analysis was conducted on 109 respondents (including 4 non-traders). The average time to complete the interview was 44.2 minutes (SD 8.7). Participants reported mean visual analogue scale (VAS) scores between 63.15 for ‘moderate impairment’ and 17.98 for ‘hand motion’ to ‘no light perception’. Mean health state utilities (HSU) varied between 0.76 (SD 0.26) in ‘moderate impairment’, and 0.20 (SD 0.58) in ‘hand motion’ to ‘no light perception’. Of all HSU evaluations, 14% were considered WTD which most commonly occurred in the most severe visually impaired health state. Conclusion This study provides valuable information on HSUs across a range of IRD health states from the Australian general population perspective. The utilities obtained in this study can be used as inputs into CUA of IRD therapies.
Suggested Citation
Maria Farris & Stephen Goodall & Richard Abreu Lourenco & Brendan Mulhern & Kathleen Manipis & Elena Meshcheriakova & Milena Lewandowska, 2024.
"Estimating Australian Population Utilities for Inherited Retinal Disease Using Time Trade-Off,"
PharmacoEconomics - Open, Springer, vol. 8(6), pages 911-922, November.
Handle:
RePEc:spr:pharmo:v:8:y:2024:i:6:d:10.1007_s41669-024-00515-5
DOI: 10.1007/s41669-024-00515-5
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