Evidence map›Paper›PMID 39102180›Full record

ArticlePharmacoEconomics - open2024

Estimating Australian Population Utilities for Inherited Retinal Disease Using Time Trade-Off.

Maria Farris, Stephen Goodall, Richard De Abreu Lourenco, Brendan Mulhern, Kathleen Manipis, Elena Meshcheriakova, Milena Lewandowska

Abstract read
In one paragraph

Article in PharmacoEconomics - open, 2024. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Not yet cited in PubMed.

0numbers the graph read from it
0cells of the map it votes in
0citing papers in PubMed
–field-weighted citation impact
1 · What the graph read from it

What it found

Each row is one number read from the abstract, on the scale the paper reported it, with its interval. Left of the dashed line favours the treatment, right favours the comparator. Under each row is the sentence it came from. New to these charts? A ten-minute tutorial.

The abstract states no effect estimate the extractor could read, or names no intervention and outcome on the map, so this paper lights no cell and moves no belief. It is still indexed, cited and linked below.

2 · The registry

The trial behind it

Trials whose registry record cites this paper, or whose number appears in the abstract. A trial that started after this paper was published is citing it as background, not reporting it.

Neither the registry nor the abstract names a trial number. If this is a trial report, that itself is worth knowing.

3 · Its place in the literature

Who cites it

0 citing papers in PubMed.

No citing paper in PubMed yet.

4 · The record

Corrections and comments

PubMed lists nothing against this paper. Absence here is not a guarantee, only a check that was made.

5 · Who and what money

Authors and funding

7 authors.

Maria FarrisNovartis Pharmaceuticals Australia, Macquarie Park, Australia. Maria.H.Kokoszka@alumni.uts.edu.au.ORCID http://orcid.org/0000-0003-3542-7162
Stephen GoodallCentre for Health Economic Research and Evaluation, University of Technology Sydney, Sydney, Australia.
Richard De Abreu LourencoCentre for Health Economic Research and Evaluation, University of Technology Sydney, Sydney, Australia.
Brendan MulhernCentre for Health Economic Research and Evaluation, University of Technology Sydney, Sydney, Australia.
Kathleen ManipisCentre for Health Economic Research and Evaluation, University of Technology Sydney, Sydney, Australia.
Elena MeshcheriakovaCentre for Health Economic Research and Evaluation, University of Technology Sydney, Sydney, Australia.
Milena LewandowskaCentre for Health Economic Research and Evaluation, University of Technology Sydney, Sydney, Australia.

Funding

Novartis Australia Industry Doctorate Program
6 · The paper itself

Abstract

purposeInherited 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.

methodsThe 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.

resultsOne 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.

conclusionThis 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.

Identifiers

PMID39102180
PMCPMC11499549

What OpenQuestion holds

Textmetadata
LicenceCC BY-NC
Read underepoch 390

Registered trials

None linked

Read under generation 80e0d062 · epoch 390. Bibliography from PubMed, PubMed Central and OpenAlex; grants from NIH RePORTER; trial links from ClinicalTrials.gov; estimates, votes and beliefs from the OpenQuestion graph.