The EQ-HWB: Overview of the Development of a Measure of Health and Wellbeing and Key Results.

OBJECTIVES: Existing measures for estimating quality-adjusted life-years are mostly limited to health-related quality of life. This article presents an overview of the development the EQ-HWB (EQ Health and Wellbeing), which is a measure that encompasses health and wellbeing. METHODS: Stages: (1) Establishing domains through reviews of the qualitative literature informed by a conceptual framework. (2) Generation and selection of items to cover the domains. (3) Face validation of these items through qualitative interviews with 168 patients, social care users, general population, and carers across 6 countries (Argentina, Australia, China, Germany, United Kingdom, United States). (4) Extensive psychometric testing of candidate items (using classical, factor analysis, and item response theory methods) on > 4000 respondents in the 6 countries. Stakeholders were consulted throughout. RESULTS: A total of 32 subdomains grouped into 7 high-level domains were identified from the qualitative literature and 97 items generated to cover them. Face validation eliminated 36 items, modified 14, and added 3. Psychometric testing of 64 items found little difference in missing data or problems with response distribution, the conceptual model was confirmed except in China, and most items performed well in the item response theory in all countries. Evidence was presented to stakeholders in 2 rounds of consultation to inform the final selection of items for the EQ-HWB (25-item) and the short version of EQ-HWB (9-items). CONCLUSIONS: EQ-HWB measures have been developed internationally for evaluating interventions in health, public health, and social care including the impact on patients, social care users, and carers.

Capturing what matters: updating NICE methods guidance on measuring and valuing health.

In July 2019, the National Institute for Health and Care Excellence (NICE) initiated a major review of its health technology evaluation methods to update its methods guide. This update has recently concluded with the publication of its health technology evaluation manual in January 2022. This paper reports the methods and findings of the review in relation to the recommended approach to use for the measurement and valuation of health-related quality of life (HRQoL) in submissions to NICE. Issues related to (i) the methods to use when NICE's preferred measure (EQ-5D) is not appropriate or not available; (ii) adjusting health state utility values over time to account for age; (iii) measuring and valuing HRQoL in children and young people; and (iv) including carers' QoL in economic evaluations were included in this review. This commentary summarises the methods used to undertake the review, its findings, and the changes to NICE methods that were proposed based on these findings. It also outlines topics where further research is needed before definitive methods guidance can be issued. The broad proposals described here were subject to a public consultation in 2020 and a further consultation on the updated methods guidance was completed in October 2021 before the publication of the manual in January 2022.

Bilateral cochlear implantation for hearing-impaired children: criterion of candidacy derived from an observational study.

OBJECTIVES: Policy-makers have struggled to define the minimum degree of hearing impairment at which children should be offered cochlear implants rather than the less invasive alternative of acoustic hearing aids. This study compared outcomes for children with bilateral cochlear implants and children with bilateral hearing aids, to determine a criterion of candidacy for pediatric bilateral cochlear implantation. DESIGN: This observational study measured the listening skills of children who received routine audiological care in the United Kingdom. Participants were recruited from hospitals, educational services, and charities. Eligibility criteria included a diagnosis of hearing impairment before 31 months of age and pure-tone thresholds greater than or equal to 50 dB HL at 2 and 4 kHz bilaterally. Seventy-one children participated, aged 46 to 86 months (mean 64 months). Twenty-eight children used bilateral implants provided in a simultaneous surgery; 43 used bilateral digital hearing aids. The two groups of children were demographically similar in variables that predict outcomes for children with hearing impairment. Children's ability to understand speech was measured using closed-set tests of word discrimination in three conditions: in quiet, in pink noise, and in two-talker babble. For each listening test, an actuarial method was used to compare the distribution of scores from children with cochlear implants and children with hearing aids. The aim was to calculate the unaided pure-tone average (PTA) hearing level at which a child has odds of 4:1 of a better outcome with implants than with hearing aids. The PTA associated with odds of 4:1 has been used previously to define criteria of candidacy for implantation. The main analyses used a four-frequency PTA (mean of unaided thresholds at 0.5, 1, 2, and 4 kHz in the better-hearing ear). Additional analyses used a three-frequency PTA (0.5, 1, and 2 kHz) and two-frequency PTA (2 and 4 kHz). RESULTS: Odds of 4:1 of a better outcome with implants were associated with a four-frequency PTA of 79, 86, and 76 dB HL for tests of word discrimination in quiet, noise, and babble, respectively. The mean of these three estimates is 80 dB HL. It can be difficult to measure a four-frequency PTA in young children, but a two-frequency PTA typically can be measured. Odds of 4:1 were associated with a two-frequency PTA of 83, 92, and 80 dB HL for tests of word discrimination in quiet, noise, and babble, respectively. The mean of these three estimates is 85 dB HL. CONCLUSIONS: Children with an unaided four-frequency PTA of 80 dB HL or poorer in both ears should be considered candidates for bilateral cochlear implantation. In cases where a four-frequency PTA cannot be measured, the criterion of candidacy should be a two-frequency PTA of 85 dB HL or poorer in both ears. If adopted by policy-makers, these recommendations would expand the provision of cochlear implants among children in England and Wales.

Test-retest reliability of the Toy Discrimination Test with a masker of noise or babble in children with hearing impairment.

OBJECTIVE: The Toy Discrimination Test measures children's ability to discriminate spoken words. Previous assessments of reliability tested children with normal hearing or mild hearing impairment, and most studies used a version of the test without a masking sound. We assessed test-retest reliability for children with hearing impairment using maskers of broadband noise and two-talker babble. DESIGN: Stimuli were presented from a loudspeaker. The signal-to-noise ratio (SNR) was varied adaptively to estimate the speech-reception threshold (SRT) corresponding to 70.7% correct performance. Participants completed each masked condition twice. STUDY SAMPLE: Fifty-five children with permanent hearing impairment participated, aged 3.0 to 6.3 years. Thirty-four children used acoustic hearing aids; 21 children used cochlear implants. RESULTS: For the noise masker, the within-subject standard deviation of SRTs was 2.4 dB, and the correlation between first and second SRT was + 0.73. For the babble masker, corresponding values were 2.7 dB and + 0.60. Reliability was similar for children with hearing aids and children with cochlear implants. CONCLUSIONS: The results can inform the interpretation of scores from individual children. If a child completes a condition twice in different listening situations (e.g. aided and unaided), a difference between scores ≥ 7.5 dB would be statistically significant (p <.05).

What is the best approach to adopt for identifying the domains for a new measure of health, social care and carer-related quality of life to measure quality-adjusted life years? Application to the development of the EQ-HWB?

Economic evaluation combines costs and benefits to support decision-making when assessing new interventions using preference-based measures to measure and value benefits in health or health-related quality of life. These health-focused instruments have limited ability to capture wider impacts on informal carers or outcomes in other sectors such as social care. Sector-specific instruments can be used but this is problematic when the impact of an intervention straddles different sectors.An alternative approach is to develop a generic preference-based measure that is sufficiently broad to capture important cross-sector outcomes. We consider the options for the selection of domains for a cross-sector generic measure including how to identify domains, who should provide information on the domains and how this should be framed. Beyond domain identification, considerations of criteria and stakeholder needs are also identified.This paper sets out the case for an approach that relies on the voice of patients, social care users and informal carers as the main source of domains and describes how the approach was operationalised in the 'Extending the QALY' project which developed the new measure, the EQ-HWB (EQ health and wellbeing instrument). We conclude by discussing the strengths and limitations of this approach. The new measure should be sufficiently generic to be used to consistently evaluate health and social care interventions, yet also sensitive enough to pick up important changes in quality of life in patients, social care users and carers.

Estimates of the cost-effectiveness of pediatric bilateral cochlear implantation.

OBJECTIVES: Objectives were, first, to estimate the additional number of quality-adjusted life years (QALYs) gained by deaf children from bilateral compared with unilateral implantation (DeltaQ); second, to estimate the additional cost to the healthcare system in the United Kingdom for providing bilateral compared with unilateral implantation (DeltaC); and, third, to compare the values of incremental net benefit (INB), rDeltaQ - DeltaC, with criteria used by policy makers in deciding whether to adopt health technologies. In England and Wales, the healthcare policy-making body must be satisfied that the INB is positive for a maximum value of r of pound30,000 (the "net-benefit" criterion). Policy makers may also require the likelihood that the technology is cost-effective to exceed 0.8 (the "likelihood" criterion). DESIGN: An opportunity sample of 180 informants, composed of clinicians/researchers, students, and parents, valued the quality of life of a hypothetical child born profoundly deaf. The child was described in written vignettes as achieving typical outcomes with no implant, a unilateral implant, a unilateral implant with benefit from a contralateral acoustic hearing aid, or bilateral implants. Valuations were made using the time trade-off (TTO) and a visual analog scale (VAS). A decision model was constructed to describe events related to implantation that could occur over a child's lifetime after the decision to implant. A cost and a probability were associated with each event. Monte Carlo simulations modeled the management of cohorts of 3000 children and estimated a value of DeltaC for each child. An increment in quality of life was sampled with replacement from the appropriate distribution of informants' valuations to estimate a value of DeltaQ for each child. The minimum value of r for which the average INB was positive was calculated to test the net-benefit criterion. The proportion of simulations for which the INB was positive when r was pound30,000 was calculated to test the likelihood criterion. RESULTS: Estimates of the cost-effectiveness of unilateral implantation aligned closely with published estimates, giving credibility to analyses of bilateral implantation. Based on TTO data (VAS data in parentheses), bilateral implantation was associated with an increment in quality of life of +0.063 (+0.076), yielding 1.57 (1.87) additional QALYs at a cost of pound34,000. Net benefit was positive, provided that pound21,768 ( pound18,173) could be spent to gain a QALY. If pound30,000 could be spent, the probability that bilateral implantation is cost-effective was 0.480 (0.539). Thus, the net-benefit criterion, but not the likelihood criterion, was met in both analyses. The net-benefit criterion was also met in analyses based on data from the three groups of informants individually. CONCLUSIONS: Groups of adults varying widely in age and life experience perceived sufficient additional quality of life from giving children two implants rather than one to mean that bilateral cochlear implantation is possibly a cost-effective use of healthcare resources in the UK. Wide variation in valuations within the groups of informants means that considerable uncertainty surrounds that conclusion. Further data on the costs and benefits of bilateral implantation are needed to resolve the uncertainty.

Use of Patient Preference Studies in HTA Decision Making: A NICE Perspective.

Patient preference studies could provide valuable insights to a National Institute for Health and Care Excellence committee into the preferences patients have for different treatment options, especially if the study sample is representative of the broader patient population. We identify three main uses of patient preference studies along a technology's pathway from drug development to clinical use: in early clinical development to guide the selection of appropriate endpoints, to inform benefit-risk assessments carried out by regulators and to inform reimbursement decisions made by health technology assessment bodies. In the context of the National Institute for Health and Care Excellence's methods and processes, we do not see a role for quantitative patient preference data to be directly incorporated into health economic modelling. Rather, we see a role for patient preference studies to be submitted alongside other types of evidence. Examples where patient preference studies might have added value in health technology assessments include cases where two distinctly different treatment options are being compared, when patients have to decide between multiple treatment options, when technologies have important non-health benefits or when a treatment is indicated for a heterogenous population.

Correction to: Use of Expert Judgement Across NICE Guidance­Making Programmes: A Review of Current Processes and Suitability of Existing Tools to Support the Use of Expert Elicitation.

In Sect. 3.3.1, the second sentence of the second paragraph.

Correction to: The Future of Precision Medicine: Potential Impacts for Health Technology Assessment.

The article The Future of Precision Medicine: Potential Impacts for Health Technology Assessment written by James Love­Koh, Alison Peel Juan, Carlos Rejon­Parrilla, KateAnastasia Chalkidou, Hannah Wood, Matthew Taylor was originally published electronically on the publisher's internet portal (currently Springer Link) on [13th July, 2018] with incorrect spelling of the co-author "Juan Carlos Rejon-Parilla". The correct spelling is "Juan Carlos Rejon-Parrilla".

Use of Expert Judgement Across NICE Guidance-Making Programmes: A Review of Current Processes and Suitability of Existing Tools to Support the Use of Expert Elicitation.

OBJECTIVES: This study aimed to review current use of experts within National Institute for Health and Care Excellence (NICE) guidance-making programmes, identify improvements in use of expert judgement, and to assess tools and protocols to support the elicitation of information from experts for use by NICE. METHODS: The study comprised a review of NICE process guides; semi-structured interviews with individuals representing each NICE guidance-making programme and a comparison of the suitability of currently available tools and protocols for expert elicitation to the requirements of NICE programmes. RESULTS: Information elicited from experts and the way in which it is used varies across NICE guidance-making programmes. Experts' involvement can be as intensive as being a member of a committee and thus having direct influence on recommendations or limited one-off consultations on specific parameters. We identified 16 tools for expert elicitation that were potentially relevant. None fully met the requirements of NICE, although an existing tool could be potentially adapted. Ongoing research to develop a reference protocol for expert elicitation in healthcare decision making may be of use in future. CONCLUSIONS: NICE uses expert judgement across all its guidance-making programmes, but its uses vary considerably. There is no currently available tool for expert elicitation suitable for use by NICE. However, adaptation of an existing tool or ongoing research in the area could address this in the future.

The Future of Precision Medicine: Potential Impacts for Health Technology Assessment.

OBJECTIVE: Precision medicine allows healthcare interventions to be tailored to groups of patients based on their disease susceptibility, diagnostic or prognostic information, or treatment response. We analysed what developments are expected in precision medicine over the next decade and considered the implications for health technology assessment (HTA) agencies. METHODS: We performed a pragmatic literature search to account for the large size and wide scope of the precision medicine literature. We refined and enriched these results with a series of expert interviews up to 1 h in length, including representatives from HTA agencies, research councils and researchers designed to cover a wide spectrum of precision medicine applications and research. RESULTS: We identified 31 relevant papers and interviewed 13 experts. We found that three types of precision medicine are expected to emerge in clinical practice: complex algorithms, digital health applications and 'omics'-based tests. These are expected to impact upon each stage of the HTA process, from scoping and modelling through to decision-making and review. The complex and uncertain treatment pathways associated with patient stratification and fast-paced technological innovation are central to these effects. DISCUSSION: Innovation in precision medicine promises substantial benefits but will change the way in which some health services are delivered and evaluated. The shelf life of guidance may decrease, structural uncertainty may increase and new equity considerations will emerge. As biomarker discovery accelerates and artificial intelligence-based technologies emerge, refinements to the methods and processes of evidence assessments will help to adapt and maintain the objective of investing in healthcare that is value for money.

The effect of early auditory experience on the spatial listening skills of children with bilateral cochlear implants.

OBJECTIVES: Both electrophysiological and behavioural studies suggest that auditory deprivation during the first months and years of life can impair listening skills. Electrophysiological studies indicate that 3½ years may be a critical age for the development of symmetrical cortical responses in children using bilateral cochlear implants. This study aimed to examine the effect of auditory experience during the first 3½ years of life on the behavioural spatial listening abilities of children using bilateral cochlear implants, with reference to normally hearing children. Data collected during research and routine clinical testing were pooled to compare the listening skills of children with bilateral cochlear implants and different periods of auditory deprivation. METHODS: Children aged 4-17 years with bilateral cochlear implants were classified into three groups. Children born profoundly deaf were in the congenital early bilateral group (received bilateral cochlear implants aged ≤3½ years, n=28) or congenital late bilateral group (received first implant aged ≤3½ years and second aged >3½ years, n=38). Children with some bilateral acoustic hearing until the age of 3½ years, who subsequently became profoundly deaf and received bilateral cochlear implants, were in the acquired/progressive group (n=16). There were 32 children in the normally hearing group. Children completed tests of sound-source localization and spatial release from masking (a measure of the ability to use both ears to understand speech in noise). RESULTS: The acquired/progressive group localized more accurately than both groups of congenitally deaf children (p<0.05). All three groups of children with cochlear implants showed similar spatial release from masking. The normally hearing group localized more accurately than all groups with bilateral cochlear implants and displayed more spatial release from masking than the congenitally deaf groups on average (p<0.05). CONCLUSION: Children with bilateral cochlear implants and early experience of acoustic hearing showed more accurate localization skills, on average, than children born profoundly deaf.

The developmental trajectory of spatial listening skills in normal-hearing children.

PURPOSE: To establish the age at which children can complete tests of spatial listening and to measure the normative relationship between age and performance. METHOD: Fifty-six normal-hearing children, ages 1.5-7.9 years, attempted tests of the ability to discriminate a sound source on the left from one on the right, to localize a source, to track moving sources, and to perceive speech in noise. RESULTS: Tests of left-right discrimination, movement tracking, and speech perception were completed by ≥ 75% of children older than 3 years. Children showed adult levels of performance from age 1.5 years (movement tracking), 3 years (left-right discrimination), and 6 years (localization and speech in noise). Spatial release from masking-calculated as the difference in speech reception thresholds between conditions with spatially coincident and spatially separate sp-eech and noise--remained constant at 5 dB from age 3 years. Data from a separate study demonstrate the age at which children with cochlear implants can complete the same tests. Assessments of left-right discrimination, movement tracking, and speech perception were completed by ≥ 75% of children who are older than 5 years and who wear cochlear implants. CONCLUSION: These data can guide the selection of tests for future studies and inform the interpretation of results from clinical populations.