Hasan Basarir

The cost-effectiveness of the Dose Adjustment for Normal Eating (DAFNE) structured education programme: an update using the Sheffield Type 1 Diabetes policy model

To estimate the cost‐effectiveness of training in flexible intensive insulin therapy [as provided in the Dose Adjustment for Normal Eating (DAFNE) structured education programme] compared with no training for adults with Type 1 diabetes mellitus in the UK using the Sheffield Type 1 Diabetes Policy Model.

Assessing the cost-effectiveness of type 1 diabetes interventions: the Sheffield type 1 diabetes policy model.

To build a flexible and comprehensive long‐term Type 1 diabetes mellitus model incorporating the most up‐to‐date methodologies to allow a number of cost‐effectiveness evaluations.

Relative effectiveness of insulin pump treatment over multiple daily injections and structured education during flexible intensive insulin treatment for type 1 diabetes: cluster randomised trial (REPOSE)

Objective To compare the effectiveness of insulin pumps with multiple daily injections for adults with type 1 diabetes, with both groups receiving equivalent training in flexible insulin treatment. Design Pragmatic, multicentre, open label, parallel group, cluster randomised controlled trial (Relative Effectiveness of Pumps Over MDI and Structured Education (REPOSE) trial). Setting Eight secondary care centres in England and Scotland. Participants Adults with type 1 diabetes who were willing to undertake intensive insulin treatment, with no preference for pumps or multiple daily injections. Participants were allocated a place on established group training courses that taught flexible intensive insulin treatment (“dose adjustment for normal eating,” DAFNE). The course groups (the clusters) were then randomly allocated in pairs to either pump or multiple daily injections. Interventions Participants attended training in flexible insulin treatment (using insulin analogues) structured around the use of pump or injections, followed for two years. Main outcome measures The primary outcomes were a change in glycated haemoglobin (HbA1c) values (%) at two years in participants with baseline HbA1c value of ≥7.5% (58 mmol/mol), and the proportion of participants achieving an HbA1c value of <7.5%. Secondary outcomes included body weight, insulin dose, and episodes of moderate and severe hypoglycaemia. Ancillary outcomes included quality of life and treatment satisfaction. Results 317 participants (46 courses) were randomised (156 pump and 161 injections). 267 attended courses and 260 were included in the intention to treat analysis, of which 235 (119 pump and 116 injection) had baseline HbA1c values of ≥7.5%. Glycaemic control and rates of severe hypoglycaemia improved in both groups. The mean change in HbA1c at two years was −0.85% with pump treatment and −0.42% with multiple daily injections. Adjusting for course, centre, age, sex, and accounting for missing values, the difference was −0.24% (−2.7 mmol/mol) in favour of pump users (95% confidence interval −0.53 to 0.05, P=0.10). Most psychosocial measures showed no difference, but pump users showed greater improvement in treatment satisfaction and some quality of life domains (dietary freedom and daily hassle) at 12 and 24 months. Conclusions Both groups showed clinically relevant and long lasting decreases in HbA1c, rates of severe hypoglycaemia, and improved psychological measures, although few participants achieved glucose levels currently recommended by national and international guidelines. Adding pump treatment to structured training in flexible intensive insulin treatment did not substantially enhance educational benefits on glycaemic control, hypoglycaemia, or psychosocial outcomes in adults with type 1 diabetes. These results do not support a policy of providing insulin pumps to adults with poor glycaemic control until the effects of training on participants’ level of engagement in intensive self management have been determined. Trial registration Current Controlled Trials ISRCTN61215213.

A Model-Based Economic Evaluation of Biologic and Non-Biologic Options for the Treatment of Adults with Moderately-to-Severely Active Ulcerative Colitis after the Failure of Conventional Therapy

BackgroundUlcerative colitis (UC) is the most common form of inflammatory bowel disease in the UK. Medical management aims to induce and maintain remission and to avoid complications and the necessity for surgical intervention. Colectomy removes the source of inflammation but is associated with morbidity and mortality. Newer anti-tumour necrosis factor (TNF)-α therapies may improve medical outcomes, albeit at an increased cost.ObjectiveOur objective was to assess the incremental cost effectiveness of infliximab, adalimumab and golimumab versus conventional therapy and surgery from a National Health Service (NHS) and Personal Social Services (PSS) perspective over a lifetime horizon.MethodsA Markov model was developed with health states defined according to whether the patient is alive or dead, current treatments received, history of colectomy and level of disease control. Transition probabilities were derived from network meta-analyses (NMAs) of trials of anti-TNF-α agents in the moderate-to-severe UC population. Health utilities, colectomy rates, surgical complications and resource use estimates were derived from literature. Unit costs were drawn from standard costing sources and literature and were valued at year 2013/2014 values.ResultsFor patients in whom surgery is an option, colectomy is expected to dominate all medical treatment options. For patients in whom colectomy is not an option, infliximab and golimumab are expected to be ruled out due to dominance, whilst the incremental cost-effectiveness ratio (ICER) for adalimumab versus conventional treatment is expected to be approximately £50,278 per quality-adjusted life-year (QALY) gained.ConclusionsBased on the NMAs, the ICERs for anti-TNF-α therapy versus conventional treatment or surgery are expected to be at best, in excess of £50,000 per QALY gained. The cost effectiveness of withdrawing biologic therapy upon remission and re-treating relapse is unknown.

Measuring quality of life in children with speech and language difficulties: a systematic review of existing approaches

BACKGROUND Childrens and adolescents speech and language difficulties (SaLD) can affect various domains of quality of life (QoL), and speech and language therapy interventions are critical to improving QoL. Systematically measuring QoL outcomes in this population is highly complex due to factors such as heterogeneity in impairments and differing targets during intervention. However, measurements of QoL are increasingly required by healthcare commissioners and policy-makers to inform resource allocation. AIMS To review the use of QoL measures in research involving children (age ≤ 18 years) with SaLD. METHODS & PROCEDURES A systematic review was undertaken. A systematic search across various databases was performed. Information on the methodological details of each relevant study, along with descriptions of the QoL measures employed, were extracted into standardized data extraction forms. Findings were discussed in a narrative synthesis. OUTCOMES & RESULTS Twenty-one relevant studies were identified that deal with a range of subpopulations of children with SaLD. For the most part, generic QoL measures were used, although there was little convergence on the type of QoL measures employed throughout the literature. Five studies utilized preference-based QoL measures, including the 16D/17D, HUI3, EQ-5D and QWB-SA. Of these measures, the HUI3 demonstrated the most promising discriminant validity, although the preference weights for this measure were generated with adults. CONCLUSIONS & IMPLICATIONS QoL among children with SaLD is not yet being captured in a systematic way. The HUI3 measure appears to show some promise for generating relevant preference-based QoL estimates, although further testing of the measure is required.

The Capability Approach: A Critical Review of Its Application in Health Economics

The capability approach is an approach to assessing well-being developed by Amartya Sen. Interest in this approach has resulted in several attempts to develop questionnaires to measure and value capability at an individual level in health economics. This commentary critically reviews the ability of these questionnaires to measure and value capability. It is argued that the method used in the questionnaires to measure capability will result in a capability set that is an inaccurate description of the individuals true capability set. The measured capability set will either represent only one combination and ignore the value of choice in the capability set, or represent one combination that is not actually achievable by the individual. In addition, existing methods of valuing capability may be inadequate because they do not consider that capability is a set. It may be practically more feasible to measure and value capability approximately rather than directly. Suggestions are made on how to measure and value an approximation to capability, but further research is required to implement the suggestions.

The Impact of Diabetes-Related Complications on Preference-Based Measures of Health-Related Quality of Life in Adults with Type I Diabetes

Introduction. This study estimates health-related quality of life (HRQoL) or utility decrements associated with type 1 diabetes mellitus (T1DM) using data from a UK research program on the Dose Adjustment For Normal Eating (DAFNE) education program. Methods. A wide range of data was collected from 2341 individuals who undertook a DAFNE course in 2009–2012, at baseline and for 2 subsequent years. We use fixed- and random-effects linear models to generate utility estimates for T1DM using different instruments: EQ-5D, SF-6D, and EQ-VAS. We show models with and without controls for HbA1c and depression, which may be endogenous (if, for example, there is reverse causality in operation). Results. We find strong evidence of an unobserved individual effect, suggesting the superiority of the fixed-effects model. Depression shows the greatest decrement across all the models in the preferred fixed-effects model. The fixed-effects EQ-5D model also finds a significant decrement from retinopathy, body mass index, and HbA1c (%). Estimating a decrement using the fixed-effects model is not possible for some conditions where there are few new cases. In the random-effects model, diabetic foot disease shows substantial utility decrements, yet these are not significant in the fixed-effects models. Conclusion. Utility decrements have been calculated for a wide variety of health states in T1DM that can be used in economic analyses. However, despite the large data set, the low incidence of several complications leads to uncertainty in calculating the utility weights. Depression and diabetic foot disease result in a substantial loss in HRQoL for patients with T1DM. HbA1c (%) appears to have an independent negative impact on HRQoL, although concerns remain regarding the potential endogeneity of this variable.

Principles of health economic evaluations of lipid-lowering strategies.

Purpose of review Policy decision-making in cardiovascular disease is increasingly informed by the results generated from decision-analytic models (DAMs). The methodological approaches and assumptions used in these DAMs impact on the results generated and can influence a policy decision based on a cost per quality-adjusted life year (QALY) threshold. Decision makers need to be provided with a clear understanding of the key sources of evidence and how they are used in the DAM to make an informed judgement on the quality and appropriateness of the results generated. Recent findings Our review identified 12 studies exploring the cost-effectiveness of pharmaceutical lipid-lowering interventions published since January 2010. All studies used Markov models with annual cycles to represent the long-term clinical pathway. Important differences in the model structures and evidence base used within the DAMs were identified. Whereas the reporting standards were reasonably good, there were many instances when reporting of methods could be improved, particularly relating to baseline risk levels, long-term benefit of treatment and health state utility values. Summary There is a scope for improvement in the reporting of evidence and modelling approaches used within DAMs to provide decision makers with a clearer understanding of the quality and validity of the results generated. This would be assisted by fuller publication of models, perhaps through detailed web appendices.

Using a Discrete-Choice Experiment Involving Cost to Value a Classification System Measuring the Quality-of-Life Impact of Self-Management for Diabetes

OBJECTIVES To describe the use of a novel approach in health valuation of a discrete-choice experiment (DCE) including a cost attribute to value a recently developed classification system for measuring the quality-of-life impact (both health and treatment experience) of self-management for diabetes. METHODS A large online survey was conducted using DCE with cost on UK respondents from the general population (n = 1497) and individuals with diabetes (n = 405). The data were modeled using a conditional logit model with robust standard errors. The marginal rate of substitution was used to generate willingness-to-pay (WTP) estimates for every state defined by the classification system. Robustness of results was assessed by including interaction effects for household income. RESULTS There were some logical inconsistencies and insignificant coefficients for the milder levels of some attributes. There were some differences in the rank ordering of different attributes for the general population and diabetic patients. The WTP to avoid the most severe state was £1118.53 per month for the general population and £2356.02 per month for the diabetic patient population. The results were largely robust. CONCLUSIONS Health and self-management can be valued in a single classification system using DCE with cost. The marginal rate of substitution for key attributes can be used to inform cost-benefit analysis of self-management interventions in diabetes using results from clinical studies in which this new classification system has been applied. The method shows promise, but found large WTP estimates exceeding the cost levels used in the survey.

Developing a questionnaire to determine the impact of self-management in diabetes: giving people with diabetes a voice

BackgroundThe prevalence of diabetes mellitus (DM) is increasing dramatically, placing considerable financial burden on the healthcare budget of each country. Patient self-management is crucial for the control of blood glucose, which largely determines the chances of developing diabetes-related complications. Self-management interventions vary widely, and a method is required for assessing the impact of self-management. This paper describes the development of a questionnaire intended for use to measure the impact of self-management in diabetes.MethodsAn iterative development process was undertaken to identify the attributes of self-management using 5 steps. First, a literature review was undertaken to identify and understand themes relating to self-management of DM to inform a topic guide. Second, the topic guide was further refined following consultation with a Patient and Public Involvement group. Third, the topic guide was used to inform semi-structured interviews with patients with Type 1 DM (T1DM) and Type 2 DM (T2DM) to identify how self-management of DM affects individuals. Fourth, the research team considered potential attributes alongside health attributes from an existing measure (Diabetes Health Profile, DHP) to produce an instrument reflecting both health and self-management outcomes simultaneously. Finally, a draft instrument was tested in a focus group to determine the wording and acceptability.ResultsSemi-structured interviews were carried out with 32 patients with T1DM and T2DM. Eight potential attributes were identified: fear/worry/anxiety, guilt, stress, stigma, hassle, control, freedom, and feeling supported. Four of these self-management attributes were selected with four health attributes (mood, worry about hypos (hypoglycaemic episodes), vitality and social limitations) to produce the Health and Self-Management in Diabetes (HASMIDv1) questionnaire.ConclusionsHASMIDv1 is a short questionnaire that contains eight items each with four response levels to measure the impact of self-management in diabetes for both T1DM and T2DM. The measure was developed using a mixed-methods approach that involved semi-structured interviews with people with diabetes. The measure has high face validity. Ongoing research is being undertaken to assess the validity of this questionnaire for measuring the impact of self-management interventions in economic evaluation.