Celia A. Brown

The stepped wedge trial design: a systematic review

BackgroundStepped wedge randomised trial designs involve sequential roll-out of an intervention to participants (individuals or clusters) over a number of time periods. By the end of the study, all participants will have received the intervention, although the order in which participants receive the intervention is determined at random. The design is particularly relevant where it is predicted that the intervention will do more good than harm (making a parallel design, in which certain participants do not receive the intervention unethical) and/or where, for logistical, practical or financial reasons, it is impossible to deliver the intervention simultaneously to all participants. Stepped wedge designs offer a number of opportunities for data analysis, particularly for modelling the effect of time on the effectiveness of an intervention. This paper presents a review of 12 studies (or protocols) that use (or plan to use) a stepped wedge design. One aim of the review is to highlight the potential for the stepped wedge design, given its infrequent use to date.MethodsComprehensive literature review of studies or protocols using a stepped wedge design. Data were extracted from the studies in three categories for subsequent consideration: study information (epidemiology, intervention, number of participants), reasons for using a stepped wedge design and methods of data analysis.ResultsThe 12 studies included in this review describe evaluations of a wide range of interventions, across different diseases in different settings. However the stepped wedge design appears to have found a niche for evaluating interventions in developing countries, specifically those concerned with HIV. There were few consistent motivations for employing a stepped wedge design or methods of data analysis across studies. The methodological descriptions of stepped wedge studies, including methods of randomisation, sample size calculations and methods of analysis, are not always complete.ConclusionWhile the stepped wedge design offers a number of opportunities for use in future evaluations, a more consistent approach to reporting and data analysis is required.

An epistemology of patient safety research: a framework for study design and interpretation. Part 2. Study design

This is the second in a four-part series of articles detailing the epistemology of patient safety research. This article concentrates on issues of study design. It first considers the range of designs that may be used in the evaluation of patient safety interventions, highlighting the circumstances in which each is appropriate. The paper then provides details about an innovative study design, the stepped wedge, which may be particularly appropriate in the context of patient safety interventions, since these are expected to do more good than harm. The unit of allocation in patient safety research is also considered, since many interventions need to be delivered at cluster or service level. The paper also discusses the need to ensure the masking of patients, caregivers, observers and analysts wherever possible to minimise information biases and the Hawthorne effect. The difficulties associated with masking in patient safety research are described and suggestions given on how these can be ameliorated. The paper finally considers the role of study design in increasing confidence in the generalisability of study results over time and place. The extent to which findings can be generalised over time and place should be considered as part of an evaluation, for example by undertaking qualitative or quantitative measures of fidelity, attitudes or subgroup effects.

Use of process measures to monitor the quality of clinical practice.

Outcomes of care are a blunt instrument for judging performance and should be replaced, say Richard J Lilford, Celia A Brown, and Jon Nicholl

An epistemology of patient safety research: a framework for study design and interpretation. Part 3. End points and measurement

This article builds on the previous two articles in this series, which focused on an evaluation framework and study designs for patient safety research. The current article focuses on what to measure as evidence of safety and how these measurements can be undertaken. It considers four different end points, highlighting their methodological advantages and disadvantages: patient outcomes, fidelity, intervening variables and clinical error. The choice of end point depends on the nature of the intervention being evaluated and the patient safety problem it has been designed to address. This paper also discusses the different methods of measuring error, reviewing best practice and paying particular attention to case note review. Two key issues with any method of data collection are ensuring construct validity and reliability. Since no end point or method of data collection is infallible, the present authors advocate the use of multiple end points and methods where feasible.

An epistemology of patient safety research: a framework for study design and interpretation. Part 1. Conceptualising and developing interventions

This is the first of a four-part series of articles examining the epistemology of patient safety research. Parts 2 and 3 will describe different study designs and methods of measuring outcomes in the evaluation of patient safety interventions, before Part 4 suggests that “one size does not fit all”. Part 1 sets the scene by defining patient safety research as a challenging form of service delivery and organisational research that has to deal (although not exclusively) with some very rare events. It then considers two inter-related ideas: a causal chain that can be used to identify where in an organisation’s structure and/or processes an intervention may impact; and the need for preimplementation evaluation of proposed interventions. Finally, the paper outlines the authors’ pragmatist ontological stance to patient safety research, which sets the philosophical basis for the remaining three articles.

Antenatal care and perinatal outcomes in Kwale district, Kenya

BackgroundThe importance of antenatal care (ANC) for improving perinatal outcomes is well established. However access to ANC in Kenya has hardly changed in the past 20 years. This study aims to identify the determinants of attending ANC and the association between attendance and behavioural and perinatal outcomes (live births and healthy birthweight) for women in the Kwale region of Kenya.MethodA Cohort survey of 1,562 perinatal outcomes (response rate 100%) during 2004–05 in the catchment areas for five Ministry of Health dispensaries in two divisions of the Kwale region. The associations between background and behavioural decisions on ANC attendance and perinatal outcomes were explored using univariate analysis and multivariate logistic regression models with backwards-stepwise elimination. The outputs from these analyses were reported as odds ratios (OR) with 95% confidence intervals (CI).ResultsOnly 32% (506/1,562) of women reported having any ANC. Women with secondary education or above (adjusted OR 1.83; 95% CI 1.06–3.15) were more likely to attend for ANC, while those living further than 5 km from a dispensary were less likely to attend (OR 0.29; 95% CI 0.22–0.39). Paradoxically, however, the number of ANC visits increased with distance from the dispensary (OR 1.46; 95% CI 1.33–1.60). Women attending ANC at least twice were more likely to have a live birth (vs. stillbirth) in both multivariate models. Women attending for two ANC visits (but not more than two) were more likely to have a healthy weight baby (OR 4.39; 95% CI 1.36–14.15).ConclusionThe low attendance for ANC, combined with a positive relationship between attendance and perinatal outcomes for the women in the Kwale region highlight the need for further research to understand reasons for attendance and non-attendance and also for strategies to be put in place to improve attendance for ANC.

An epistemology of patient safety research: a framework for study design and interpretation. Part 4. One size does not fit all

This is the final article in the series on the epistemology of patient safety research, and considers the selection of study design and end points during the planning of an evaluation. The key message of this series is that “one size does not fit all”: the nature of the evaluation will depend on logistical and pragmatic constraints, a priori assessment of the probability of benefits and harms, the plausible scale of these effects and the target audience for the results. This paper also discusses the advantages of mixed method designs. The strength of any conclusions can be increased if different end points concur and the authors therefore advocate assessment of the effect of the intervention on different end points across the generic causal chain linking structure, process and outcome. The use of both qualitative and quantitative methods is also advocated to help explain findings, generate theory and help contextualise results. We show how a bayesian framework can be used to synthesise evidence from a number of different sources and why this approach may be particularly appropriate for the evaluation of patient safety interventions.

Evaluating service delivery interventions to enhance patient safety

Celia Brown and Richard Lilford describe the recommendations of an MRC sponsored network to improve the quality of research into patient safety

Early‐Stage Valuation of Medical Devices: The Role of Developmental Uncertainty

BACKGROUND At the concept stage, many uncertainties surround the commercial viability of a new medical device. These include the ultimate functionality of the device, the cost of producing it and whether, and at what price, it can be sold to a health-care provider (HCP). Simple assessments of value can be made by estimating such unknowns, but the levels of uncertainty may mean that their operational value for investment decisions is unclear. However, many decisions taken at the concept stage are reversible and will be reconsidered later before the product is brought to market. This flexibility can be exploited to enhance early-stage valuations. OBJECTIVES To develop a framework for valuing a new medical device at the concept stage that balances benefit to the HCP against commercial costs. This is done within a simplified stage-gated model of the development cycle for new products. The approach is intended to complement existing proposals for the evaluation of the commercial headroom available to new medical products. CONCLUSIONS A model based on two decision gates can lead to lower bounds (underestimates) for product value that can serve to support a decision to develop the product. Quantifiable uncertainty that can be resolved before the device is brought to market will generally enhance early-stage valuations of the device, and this remains true even when some components of uncertainty cannot be fully described. Clinical trials and other evidence-gathering activities undertaken as part of the development process can contribute to early-stage estimates of value.

Selecting medical students

Tests of cognitive ability are probably the best method at present