Dataset Information

Time Trade-Off Utilities for Hereditary Angioedema Health and Caregiver States.

ABSTRACT:

Background

Hereditary angioedema (HAE) is an inherited chronic rare disease characterised by recurrent swelling attacks that are associated with significant physical and psychological burden. There is limited understanding of the effect of attack location on this burden and of caregiver burden.

Objective

Our objective was to capture the relative burden of HAE health and caregiver states, including different attack locations, through a time trade-off (TTO) analysis involving participants from the general public.

Methods

Qualitative interviews were undertaken to inform vignette development for the TTO study, including vignettes for abdominal, facial, hand and laryngeal attack health states, and an attack-free and caregiver state. Members of the general public in England rated vignettes in TTO interviews, which included a visual analogue scale (VAS) component. For the development of the health state vignettes, qualitative interviews with 15 patients, 5 caregivers and 1 clinical expert were performed. TTO analysis was based on vignette valuation completed by 100 members of the general public.

Results

The TTO values were as follows: attack-free, 0.783 (standard deviation [SD] 0.316); hand: 0.582 (SD 0.380); facial: 0.483 (SD 0.448); abdominal: 0.345 (SD 0.458); and laryngeal: 0.128 (SD 0.529). The caregiver rating was 0.762 (SD 0.303). V' scores were similar and consistent with TTO values.

Conclusion

TTO utility values demonstrate that HAE places a significant burden on patients, which is influenced by attack location, and on caregivers. These utility weights can provide important information on quality of life for future economic evaluations of treatments.

SUBMITTER: Lo SH

PROVIDER: S-EPMC8864034 | biostudies-literature | 2022 Mar

REPOSITORIES: biostudies-literature

ACCESS DATA

Publications

Time Trade-Off Utilities for Hereditary Angioedema Health and Caregiver States.

Lo Siu Hing SH Lloyd Andrew A Elkhalifa Shuayb S Sisic Zlatko Z van Nooten Floortje E FE

PharmacoEconomics - open 20210917 2

<h4>Background</h4>Hereditary angioedema (HAE) is an inherited chronic rare disease characterised by recurrent swelling attacks that are associated with significant physical and psychological burden. There is limited understanding of the effect of attack location on this burden and of caregiver burden.<h4>Objective</h4>Our objective was to capture the relative burden of HAE health and caregiver states, including different attack locations, through a time trade-off (TTO) analysis involving partic ...[more]

PMID: 34532843

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Project description: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.

Project description:BackgroundDermatology Life Quality Index (DLQI) scores are used in many countries as access and reimbursement criteria for costly dermatological treatments. In this study we examined how time trade-off (TTO) utility valuations made by individuals from the general population are related to combinations of DLQI severity levels characterizing dermatologically relevant health states, with the ultimate purpose of developing a value set for the DLQI.MethodsWe used data from an online cross-sectional survey conducted in Hungary in 2020 (n = 842 after sample exclusions). Respondents were assigned to one of 18 random blocks and were asked to provide 10-year TTO valuations for the corresponding five hypothetical health states. To analyze the relationship between DLQI severity levels and utility valuations, we estimated linear, censored, ordinal, and beta regression models, complemented by two-part scalable models accommodating heterogeneity effects in respondents' valuation scale usage. Successive severity levels (0-3) of each DLQI item were represented by dummy variables. We used cross-validation methods to reduce the initial set of 30 dummy variables and improve model robustness.ResultsOur final, censored linear regression model with 13 dummy variables had R2 = 0.136, thus accounting for 36.9% of the incremental explanatory power of a maximal (full-information) benchmark model (R2 = 0.148) over the uni-dimensional model (R2 = 0.129). Each DLQI item was found to have a negative effect on the valuation of health states, yet this effect was largely heterogeneous across DLQI items, and the relative contribution of distinctive severity levels also varied substantially. Overall, we found that the social/interpersonal consequences of skin conditions (in the areas of social and leisure activities, work and school, close personal relationships, and sexuality) had roughly twice as large disutility impact as the physical/practical aspects.ConclusionsWe have developed an experimental value set for the DLQI, which could prospectively be used for quantifying the quality-adjusted life years impact of dermatological treatments and serve as a basis for cost-effectiveness analyses. We suggest that, after validation of our main results through confirmatory studies, population-specific DLQI value sets could be developed and used for conducting cost-effectiveness analyses and developing financing guidelines in dermatological care.

Dataset Information

Time Trade-Off Utilities for Hereditary Angioedema Health and Caregiver States.

Background

Objective

Methods

Results

Conclusion

Publications

Time Trade-Off Utilities for Hereditary Angioedema Health and Caregiver States.

Similar Datasets

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