Paula Lorgelly

A qualitative assessment of the content validity of the ICECAP-A and EQ-5D-5L and their appropriateness for use in health research.

Purpose The ICECAP-A and EQ-5D-5L are two index measures appropriate for use in health research. Assessment of content validity allows understanding of whether a measure captures the most relevant and important aspects of a concept. This paper reports a qualitative assessment of the content validity and appropriateness for use of the eq-5D-5L and ICECAP-A measures, using novel methodology. Methods In-depth semi-structured interviews were conducted with research professionals in the UK and Australia. Informants were purposively sampled based on their professional role. Data were analysed in an iterative, thematic and constant comparative manner. A two stage investigation - the comparative direct approach - was developed to address the methodological challenges of the content validity research and allow rigorous assessment. Results Informants viewed the ICECAP-A as an assessment of the broader determinants of quality of life, but lacking in assessment of health-related determinants. The eq-5D-5L was viewed as offering good coverage of health determinants, but as lacking in assessment of these broader determinants. Informants held some concerns about the content or wording of the Self-care, Pain/Discomfort and Anxiety/Depression items (EQ-5D-5L) and the Enjoyment, Achievement and attachment items (ICECAP-A). Conclusion Using rigorous qualitative methodology the results suggest that the ICECAP-A and EQ-5D-5L hold acceptable levels of content validity and are appropriate for use in health research. This work adds expert opinion to the emerging body of research using patients and public to validate these measures.

Anchoring vignettes for health comparisons: an analysis of response consistency

PurposeSelf-rated health (SRH) is widely used to measure and compare the health status of different groups of individuals. However, SRH can suffer from heterogeneity in reporting styles, making health comparisons problematic. Anchoring vignettes is a promising technique for improving inter-group comparisons of SRH. A key identifying assumption of the approach is response consistency—that respondents rate themselves using the same underlying response scale that they rate the vignettes. Despite growing research into response consistency, it remains unclear how respondents rate vignettes and why respondents may not assess vignettes and themselves consistently.MethodVignettes for the EQ-5D-5L were developed and included in an online survey. In-depth interviews were conducted with participants following survey completion. Response consistency was examined through qualitative analysis of the interview responses and quantitative coding of participants’ thought processes.ResultsOur analysis showed that anchoring vignettes for the EQ-5D-5L is feasible, but that response consistency may not hold for some participants. Respondents are more likely to rate their own health and vignettes in the same way if presented with overall health state vignettes than single health dimension vignettes, and if they imagined themselves in the health state of the hypothetical individual.ConclusionThis research highlights opportunities to improve the design of anchoring vignettes in order to enhance response consistency. It additionally provides new evidence on the feasibility of employing anchoring vignettes for the EQ-5D-5L, which is promising for future work to address reporting heterogeneity in the EQ-5D-5L.

Choice of Outcome Measure in an Economic Evaluation: A Potential Role for the Capability Approach.

The last decade has seen a renewed interest in Sen’s capability approach; health economists have been instrumental in leading much of this work. One particular stream of research is the application of the approach to outcome measurement. To date, there have been a dozen attempts (some combined) to operationalise the approach, and produce an outcome measure that offers a broader evaluative space than health-related quality-of-life measures. Applications have so far been confined to public health, physical, mental health and social care interventions, but the capability approach could be of benefit to evaluations of pharmacotherapies and other technologies. This paper provides an introduction to the capability approach, reviews the measures that are available for use in an economic evaluation, including their current applications, and then concludes with a discussion of a number of issues that require further consideration before the approach is adopted more widely to inform resource allocation decisions.

Outcomes in Economic Evaluations of Public Health Interventions in Low- and Middle-Income Countries: Health, Capabilities and Subjective Wellbeing

Abstract Public health programmes tend to be complex and may combine social strategies with aspects of empowerment, capacity building and knowledge across sectors. The nature of the programmes means that some effects are likely to occur outside the healthcare sector; this breadth impacts on the choice of health and non‐health outcomes to measure and value in an economic evaluation. Employing conventional outcome measures in evaluations of public health has been questioned. There are concerns that such measures are too narrow, overlook important dimensions of programme effect and, thus, lead to such interventions being undervalued. This issue is of particular importance for low‐income and middle‐income countries, which face considerable budget constraints, yet deliver a large proportion of health activities within public health programmes. The need to develop outcome measures, which include broader measures of quality of life, has given impetus to the development of a variety of new, holistic approaches, including Sens capability framework and measures of subjective wellbeing. Despite their promise, these approaches have not yet been widely applied, perhaps because they present significant methodological challenges. This paper outlines the methodological challenges for the identification and measurement of broader outcomes of public health interventions in economic evaluation in low‐income and middle‐income countries.

The "Hazards" of Extrapolating Survival Curves

Background. It is widely recommended that health technology appraisals adopt a lifetime horizon to assess the relative costs and benefits of an intervention. However, most trials or clinical studies have relatively short follow-up periods, with the event of interest not occurring before the end of the study for many subjects. In such cases, survival analysis using parametric models can be used to extrapolate into the future. Objective. To assess the accuracy of survival analysis in projecting future events beyond the sample estimation period. Design. Using a previously published comparison of 2 alternative hip replacement prostheses based on 8 years of data as a case study, we extend the data set to include 8 years more data. Using the new data, the parametric assumptions of the previous study and its success in predicting the outcomes are assessed. Results. The extended data set casts doubt on the previous study’s findings. The failure curves of the 2 prostheses now cross, and the proportional hazards assumption no longer holds. Extrapolations from the original data set yielded very good predictions for one prosthesis for the full 16 years but were much poorer for the other, even when the proportionality assumption was relaxed. Conclusions. Care should be taken when extrapolating treatment benefits for new technologies early in their life cycle based on observational or randomized controlled trial data sources. This case study reveals that predictions of prosthesis failure based on a short follow-up period were inaccurate compared with those after a longer period of follow-up.

Can choices between alternative hip prostheses be evidence based? a review of the economic evaluation literature

BackgroundTotal hip replacement surgery places a considerable financial burden on health services and society. Given the large number of hip prostheses available to surgeons, reliable economic evidence is crucial to inform resource allocation decisions. This review summarises published economic evidence on alternative hip prostheses to examine the potential for the literature to inform resource allocation decisions in the UK.MethodsWe searched nine medical and economics electronic databases. 3,270 studies were initially identified, 17 studies were included in the review. Studies were critically appraised using three separate guidelines.ResultsSeveral methodological problems were identified including a lack of observed long term prosthesis survival data, limited up-to-date and UK based evidence and exclusion of patient and societal perspectives.ConclusionsMore clinical trials including long term follow-up and economic evaluation are needed. These should compare the cost-effectiveness of different prostheses with longer-term follow-up and including a wider perspective.

A cluster randomised controlled trial of a brief couple-focused psychoeducational intervention to prevent common postnatal mental disorders among women: study protocol.

Introduction Postnatal common mental disorders among women are an important public health problem internationally. Interventions to prevent postnatal depression have had limited success. What Were We Thinking (WWWT) is a structured, gender-informed, psychoeducational group programme for parents and their first infant that addresses two modifiable risks to postnatal mental health. This paper describes the protocol for a cluster randomised controlled trial to test the clinical effectiveness and cost-effectiveness of WWWT when implemented in usual primary care. Methods and analysis 48 maternal and child health (MCH) centres from six diverse Local Government Areas, in Victoria, Australia are randomly allocated to the intervention group (usual care plus WWWT) or the control group (usual care). The required sample size is 184 women in each group. English-speaking primiparous women receiving postpartum healthcare in participating MCH centres complete two computer-assisted telephone interviews: baseline at 4 weeks and outcome at 6 months postpartum. Women attending intervention MCH centres are invited to attend WWWT in addition to usual care. The primary outcome is meeting Diagnostic and Statistical Manual-IV (DSM-IV) diagnostic criteria for major depressive episode; generalised anxiety disorder; panic disorder with or without agoraphobia, agoraphobia with or without panic, social phobia, adult separation anxiety or adjustment disorder with depressed mood, anxiety or mixed depressed mood and anxiety within the past 30 days at 6 months postpartum. Secondary outcomes are self-rated general and emotional health, infant sleep problems, method of infant feeding, quality of mother–infant relationship and intimate partner relationship, and healthcare costs and outcomes. Ethics and dissemination Approval to conduct the study has been granted. A comprehensive dissemination plan has been devised. Trial registration number Australian New Zealand Clinical Trials Registry ACTRN12613000506796. UTN U1111-1125-8208.

THE EFFECTIvENESS OF A MUlTIDISCIplINARy FOOT CARE pROgRAM FOR CHIlDREN AND ADOlESCENTS wITH JUvENIlE IDIOpATHIC ARTHRITIS: AN EXplORATORy TRIAl

OBJECTIVES To evaluate the effectiveness of multidisciplinary foot-care, and to evaluate the methodological considerations of a trial of multidisciplinary care in juvenile idiopathic arthritis. DESIGN Exploratory randomised controlled trial. SUBJECTS/PATIENTS Children/adolescents with juvenile idio-pathic arthritis and inflammatory joint disease affecting the foot/ankle. METHODS Standard medical care was compared with a 12 month program of multidisciplinary foot-care informed by musculoskeletal ultrasound. This program was centred on strict disease control through rigorous examination and interventions delivered by a team comprised of a paediatric rheumatologist, podiatrist, physiotherapist and musculoskeletal ultrasonographer. Patients were assessed on foot impairment and disability scores using the Juvenile Arthritis Foot Disability Index. RESULTS Forty-four participants, aged 3-17 years were randomly assigned to receive the experimental (n = 21) or usual care (n = 23) interventions. There was an overall improvement in levels of foot related impairments in both groups over 12 months. Between-group differences in change scores for the Juvenile Arthritis Foot Disability Index were not statistically significant at 6 or 12 month follow-ups. CONCLUSION The integrated multidisciplinary foot care interventions described in this trial were safe, but did not improve foot impairment levels relative to usual care. This trial identified several methodological challenges including recruitment/retention, difficulties with outcome tools and potential confounders.

Gender-informed, psychoeducational programme for couples to prevent postnatal common mental disorders among primiparous women: cluster randomised controlled trial

Objectives Interventions to prevent postpartum common mental disorders (PCMD) among unselected populations of women have had limited success. The aim was to determine whether What Were We Thinking (WWWT) a gender-informed, psychoeducational programme for couples and babies can prevent PCMD among primiparous women 6 months postpartum. Design Cluster-randomised controlled trial. Setting 48 Maternal and Child Health Centres (MCHCs) from 6 Local Government Areas in Melbourne, Australia were allocated randomly to usual care (24) or usual care plus WWWT (24). Participants English-speaking primiparous women receiving primary care at trial MCHCs were recruited to the intervention (204) and control (196) conditions. Of these, 187 (91.7%) and 177 (90.3%) provided complete data. Intervention WWWT is a manualised programme comprising primary care from a trained nurse, print materials and a face-to-face seminar. Main outcome measures Data sources were standardised and study-specific measures collected in blinded computer-assisted telephone interviews at 6 and 26 weeks postpartum. The primary outcome was PCMD assessed by Composite International Diagnostic Interviews and Patient Health Questionnaire (PHQ) Depression and Generalised Anxiety Disorder modules. Results In intention-to-treat analyses the adjusted OR (AOR) of PCMD in the intervention compared to the usual care group was 0.78 (95% CI 0.38 to 1.63, ns), but mild to moderate anxiety symptoms (AOR 0.58, 95% CI 0.35 to 0.97) and poor self-rated health (AOR 0.46, 95% CI 0.22 to 0.97) were significantly lower. In a per protocol analysis, comparing the full (three component) intervention and usual care groups, the AOR of PCMD was 0.36, (95% CI 0.14 to 0.95). The WWWT seminar was appraised as salient, comprehensible and useful by >85% participants. No harms were detected. Conclusions WWWT is readily integrated into primary care, enables inclusion of fathers and addresses modifiable risks for PCMD directly. The full intervention appears a promising programme for preventing PCMD, optimising family functioning, and as the first component of a stepped approach to mental healthcare. Trial registration number ACTRN12613000506796; Results.

A protocol for a discrete choice experiment: understanding preferences of patients with cancer towards their cancer care across metropolitan and rural regions in Australia

Introduction Medical decision-making in oncology is a complicated process and to date there are few studies examining how patients with cancer make choices with respect to different features of their care. It is also unknown whether patient choices vary by geographical location and how location could account for observed rural and metropolitan cancer differences. This paper describes an ongoing study that aims to (1) examine patient and healthcare-related factors that influence choices of patients with cancer; (2) measure and quantify preferences of patients with cancer towards cancer care using a discrete choice experiment (DCE) and (3) explore preference heterogeneity between metropolitan and rural locations. Methods and analysis A DCE is being conducted to understand how patients with cancer choose between two clinical scenarios accounting for different patient and healthcare-related factors (and levels). Preliminary qualitative research was undertaken to guide the development of an appropriate DCE design including characteristics that are important and relevant to patients with cancer. A fractional factorial design using the D-efficiency criteria was used to estimate interactions among attributes. Multinomial logistic regression will be used for the primary DCE analysis and to control for sociodemographic and clinical characteristics. Ethics and dissemination The Barwon Health Human Research Ethics Committee approved the study. Findings from the study will be presented in national/international conferences and peer-reviewed journals. Our results will form the basis of a feasibility study to inform the development of a larger scale study into preferences of patients with cancer and their association with cancer outcomes.