This series is intended to promote discussion papers in progress. The Views expressed are those of the authors, and therefore should not be quoted without their permission. However, comments are welcome and we ask that they be sent direct to the corresponding author.
To view the full text documents you will need Acrobat Reader. If you do not have it, it can be downloaded free from Adobe Acrobat Reader.
Use the drop-down menu to search by year, and the search box to enter keywords or an author's surname.
For all older discussion papers please click here.
For HEDS staff please click here to download the process for the HEDS Discussion Paper Series.
This document was prepared as a pre-read paper for people attending an international workshop convened by Bellberry, Ltd. The workshop was held to discuss the challenges in valuing and paying for combination regimens in oncology. Cancers often arise through multiple biological mechanisms, meaning that treatments that target only one of these mechanisms may not provide long lasting benefit to patients. Therefore, it is common to treat cancers with multiple treatments, called combination therapies. Combination therapies are often expensive, especially when multiple on-patent treatments are combined, which makes paying for them challenging for health systems around the world. This is particularly the case when different companies own the on-patent treatments that are combined, because this may inhibit flexibility in pricing. Hence, this report primarily considers the case where combination therapies consist of two or more on-patent treatments, owned by two or more companies.
N Latimer, D Pollard
Following a conceptual modelling exercise and a set of rapid literature reviews, an economic model was developed in MS-Excel to determine whether or not the eRAT was likely to be cost-effective based on data from the ECASS trial and from the literature. The baseline population represented a cohort of patients presenting to the GP with symptoms of OG cancer. The intervention arm represented GP practices where the eRAT had been installed and training carried out, whereas the comparator arm represented practices without eRAT. Incremental outcomes were modelled using probabilistic sensitivity analysis to enable uncertainty to be estimated. A maximum justifiable cost analysis was carried out to estimate what the maximum cost of eRAT installation and training could be whilst still allowing the eRAT to be cost-effective.
Tushar Srivastava, Chloe Thomas, Duncan Chambers, Sophie Whyte
Modelling alcohol use behavior at population scale based on social role theory: An exploratory agent-based model
The relationships between social roles and patterns of alcohol use have been explored empirically within the alcohol research community; however, the mechanisms by which social roles impact on alcohol consumption, and vice versa, remain poorly theorized. We present a set of mechanisms based on social role theory to model the interaction of social roles with alcohol use behaviors at population-level.
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