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Patient reported outcome measures of quality of life in Duchenne muscular dystrophy (DMD): a systematic review of content and structural validity using COSMIN
Duchenne muscular dystrophy (DMD) is an inherited neuromuscular disorder that predominantly affects boys and men. There is no known cure, so current clinical efforts are focused on improving the health-related quality of life (QoL) of people with DMD. A number of PROMs have been used to assess QoL in people with DMD in the absence of any robust evidence base. In the current report we critically reviewed the content and structural validity of PROMs used to assess QoL in people with DMD, using robust, updated Consensus-based Standards for the selection of health Measurement INstruments (COSMIN) guidelines.
Two-stage estimation to adjust for treatment switching in randomised trials: A simulation study investigating the use of inverse probability weighting instead of re-censoring
Treatment switching is common in randomised trials of oncology treatments, with control group patients switching onto the experimental treatment during follow-up. Two-stage estimation (TSE) can be used to estimate counterfactual survival times for patients who switch treatments – that is, survival times that would have been observed in the absence of switching. However, re-censoring is usually applied alongside TSE to avoid informative censoring, but this results in lost longer-term information. We aim to determine whether combining TSE with inverse probability of censoring weights (IPCW) represents a valid alternative to re-censoring.
Assessment of the psychometric properties and refinement of the Health and Self-Management in Diabetes Questionnaire (HASMID)
The Health And Self-Management In Diabetes (HASMIDv1) questionnaire consists of eight attributes, four about quality of life, and four about self-management. The overall aim of this study was to rigorously examine the psychometric properties of the HASMIDv1 questionnaire. The study had two phases: Phase 1 concerned the identification of any items of the HASMIDv1 questionnaire that may require rewording and deriving alternative and any additional items; Phase 2 concerned the assessment of the psychometric properties of the HASMIDv1 questionnaire in a large observational survey of people with diabetes using two methods of administration (online and postal).
This discussion paper describes initial findings of a qualitative study exploring patients’ experiences (including signs, symptoms and impacts) of living with five common vascular diseases; peripheral arterial disease, carotid artery disease, abdominal aortic aneurysm, varicose veins and venous leg ulcers.
This study provides a unique overview of the five conditions, and has allowed for the similarities and differences of patient experiences to be explored. The findings are relevant to both clinicians and the NHS, and have provided a starting point for the development of a PROM for use with vascular patients.
R Duncan, E Lumley, A Tod, J Hughes, S Palfreyman, G Jones, S Nawaz, J Michaels
Optimising the cost effectiveness of repeated FIT screening and screening strategies combining bowel scope and FIT screening
A ScHARR report commissioned by the UK National Screening Committee (NSC) to consider the cost-effectiveness and endoscopy capacity requirements of a variety of different screening options incorporating faecal immunochemical testing (FIT) and bowel scope (BS) within the Bowel Cancer Screening Programme (BCSP).
S Whyte, C Thomas, B Kearns, M Webster, J Chilcott
Rapid reviews are of increasing importance within health technology assessment (HTA) due to the need for timely evidence to underpin the assessment of new technologies. A team in ScHARR have developed a decision tool for reviewers to use to select the most appropriate rapid review approaches for HTA. This paper presents the protocol for a Delphi study to validate the decision tool.
Eva Kaltenthaler, Katy Cooper, Marrissa Martyn St James, Abdullah Pandor, Ruth Wong
This paper presents two studies exploring the latent structure of item sets used in the development of the Recovering Quality of Life mental health outcome measures: ReQoL-10 and ReQoL-20.
A Keetharuth, M Barkham, J B Bjorner, J Browne, T Croudace, and J Brazier
Integrating Qualitative and Quantitative Data in the Development of Outcome Measures: The Case of the Recovering Quality of Life (ReQoL) Measures in Mental Health Populations
This paper reports in detail a novel approach of combining qualitative and quantitative evidence that was used in the development of the Recovering Quality of Life (ReQoL) measures.
A Keetharuth, E Taylor Buck, C Acquadro, K Conway, J Connell, M Barkham, J Carlton, T Ricketts, R Barber and J Brazier
The importance of content and face validity in instrument development: Lessons learnt from service users when developing the Recovering Quality of Life Measure (ReQoL)
This paper emphasises the importance of conducting face validity interviews with service users and reports the key themes of what should be considered in measure development using the development of the Recovering Quality of Life (ReQoL) measures as an example. This is often a stage in measure development that is not reported in detail.
J Connell, J Carlton, A Grundy, E Taylor Buck, A Keetharuth, T Ricketts, M Barkham, D Robotham, D Rose, and J Brazier
The "Reappraisal of the options for colorectal cancer screening report" is a substantial update to the "Option appraisal of population-based colorectal cancer screening programmes in England" (Tappenden et al. 2007). It includes a significant development in modelling methodology, and incorporation of data from (1)the first two rounds of the England gFOBT BCSP, (2) a large randomized UK trial of FS for ages 55 to 64 years, and (3) further data on the immunochemical FOBT (iFOBT).
S Whyte, J Chilcott, K Cooper, M Essat, J Stevens, R Wong, N Kalita
Causal inference for long-term survival in randomised trials with treatment switching: Should re-censoring be applied when estimating counterfactual survival times?
In this paper we present a simulation study designed to investigate applications of statistical methods used to adjust for treatment switching with and without re-censoring, to determine whether re-censoring should always be recommended within adjustment analyses. This investigates the context typically seen in health technology assessment, where estimates of long-term treatment effects are required.
N Latimer, I White, K Abrams, U Siebert
Estimating a Dutch value set for the paediatric preference-based CHU-9D using a discrete choice experiment with duration
This paper presents the development of the Dutch value set for the CHU-9D, a paediatric preference-based measure of quality of life that can be used to generate quality adjusted life years (QALYs). A large online survey was conducted using a discrete choice experiment (DCE) including a duration attribute with adult members of the Netherlands general population (n=1,276). Normative questions are raised around the valuation of paediatric preference-based measures including the appropriate perspective for imagining hypothetical paediatric health states.
D Rowen, B Mulhern, K Stevens, E Vermaire
The prevalence of diabetes mellitus (DM) is increasing dramatically and patient self-management is crucial for the control of blood glucose levels. 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.
J Carlton, J Elliott, D Rowen, K Stevens, H Basarir, K Meadows, J Brazier
This paper generates health state utility values for a novel classification system measuring the quality of life impact of self-management for diabetes, that can be used to generate Quality Adjusted Life Years (QALYs). A large online survey was conducted using a discrete choice experiment (DCE) with duration as an additional attribute on members of the UK general population (n=1,493) to elicit values for health (social limitations, mood, vitality, hypoglycaemia) and non-health (stress, hassle, control, support) aspects of self-management in diabetes. The results presented in the paper can be used in economic models of the cost-effectiveness of new interventions to value the day to day impact of the interventions for health and self-management of diabetes.
D Rowen, A Labeit, K Stevens, J Elliott, B Mulhern, J Carlton, H Basarir, J Brazier
This paper describes 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. A large online survey was conducted using DCE with cost on UK respondents from the general population (n=1,497) and individuals with diabetes (n=405). The approach shows promise, but found large willingness to pay estimates exceeding the cost levels used in the survey.
D Rowen, A Labeit, K Stevens, J Elliott, B Mulhern, J Carlton, H Basarir, J Brazier