Andrew Cook

A systematic review of models to predict recruitment to multicentre clinical trials

BackgroundLess than one third of publicly funded trials managed to recruit according to their original plan often resulting in request for additional funding and/or time extensions. The aim was to identify models which might be useful to a major public funder of randomised controlled trials when estimating likely time requirements for recruiting trial participants. The requirements of a useful model were identified as usability, based on experience, able to reflect time trends, accounting for centre recruitment and contribution to a commissioning decision.MethodsA systematic review of English language articles using MEDLINE and EMBASE. Search terms included: randomised controlled trial, patient, accrual, predict, enrol, models, statistical; Bayes Theorem; Decision Theory; Monte Carlo Method and Poisson. Only studies discussing prediction of recruitment to trials using a modelling approach were included. Information was extracted from articles by one author, and checked by a second, using a pre-defined form.ResultsOut of 326 identified abstracts, only 8 met all the inclusion criteria. Of these 8 studies examined, there are five major classes of model discussed: the unconditional model, the conditional model, the Poisson model, Bayesian models and Monte Carlo simulation of Markov models. None of these meet all the pre-identified needs of the funder.ConclusionsTo meet the needs of a number of research programmes, a new model is required as a matter of importance. Any model chosen should be validated against both retrospective and prospective data, to ensure the predictions it gives are superior to those currently used.

Use of a modified Delphi approach to develop research priorities for the Association of Coloproctology of Great Britain and Ireland

The modified Delphi approach is an established method for reaching a consensus opinion among a group of experts in a particular field. We have used this technique to survey the entire membership of the Association of Coloproctology of Great Britain and Ireland (ACPGBI) to reach a consensus on prioritizing clinical research questions in colorectal disease.

Publication rate for funded studies from a major UK health research funder: a cohort study

Objectives This study aimed to investigate what percentage of National Institute for Health Research (NIHR) Health Technology Assessment (HTA) Programme-funded projects have published their final reports in the programmes journal HTA and to explore reasons for non-publication. Design Retrospective cohort study. Setting Failure to publish findings from research is a significant area of research waste. It has previously been suggested that potentially over 50% of studies funded are never published. Participants All NIHR HTA projects with a planned submission date for their final report for publication in the journal series on or before 9 December 2011 were included. Primary and secondary outcome measures The projects were classified according to the type of research, whether they had been published or not; if not yet published, whether they would be published in the future or not. The reasons for non-publication were investigated. Results 628 projects were included: 582 (92.7%) had published a monograph; 19 (3%) were expected to publish a monograph; 13 (2.1%) were discontinued studies and would not publish; 12 (1.9%) submitted a report which did not lead to a publication as a monograph; and two (0.3%) did not submit a report. Overall, 95.7% of HTA studies either have published or will publish a monograph: 94% for those commissioned in 2002 or before and 98% for those commissioned after 2002. Of the 27 projects for which there will be no report, the majority (21) were commissioned in 2002 or before. Reasons why projects failed to complete included failure to recruit; issues concerning the organisation where the research was taking place; drug licensing issues; staffing issues; and access to data. Conclusions The percentage of HTA projects for which a monograph is published is high. The advantages of funding organisations requiring publication in their own journal include avoidance of publication bias and research waste.

The health technology assessment adaptation toolkit: Description and use

OBJECTIVES Adapting health technology assessment (HTA) reports for different contexts could reduce the need for multiple reports on the same health technology with resultant saving of time and resources. This article describes an instrument, the adaptation toolkit, which has been developed to aid in the process of adaptation of HTA reports. METHODS The toolkit was developed by a partnership of HTA agencies and networks from across Europe. The role of the toolkit is to guide the user through the process of selecting possible relevant material from these report(s), assessing the relevance, reliability, and transferability of the material, and adapting it for the desired context. RESULTS The adaptation toolkit has been developed, it comprises a collection of resources that help the user assess whether data and information in existing HTA reports should and could be adapted for their own setting. The toolkit contains two sections: a preliminary speedy sifting section and the main toolkit. The main toolkit includes five domains: (i) technology use and development, (ii) safety, (iii) effectiveness (including efficacy), (iv) economic evaluation, and (v) organizational aspects. Legal, ethical, and social aspects are beyond the scope of the toolkit. The toolkit is designed for the adaptation of evidence synthesis rather than primary research. CONCLUSIONS The completed current version of the toolkit contains checklists and resources to aid in the adaptation of HTA reports. This collection of resources is available for use by all HTA agencies and can be accessed at: http://www.eunethta.net/upload/WP5/EUnetHTA_HTA_Adaptation_Toolkit_October08.pdf..

Descriptions of non-pharmacological interventions in clinical trials

Reporting must improve

The Southampton Consensus Guidelines for Laparoscopic Liver Surgery: From Indication to Implementation

Objective: The European Guidelines Meeting on Laparoscopic Liver Surgery was held in Southampton on February 10 and 11, 2017 with the aim of presenting and validating clinical practice guidelines for laparoscopic liver surgery. Background: The exponential growth of laparoscopic liver surgery in recent years mandates the development of clinical practice guidelines to direct the specialitys continued safe progression and dissemination. Methods: A unique approach to the development of clinical guidelines was adopted. Three well-validated methods were integrated: the Scottish Intercollegiate Guidelines Network methodology for the assessment of evidence and development of guideline statements; the Delphi method of establishing expert consensus, and the AGREE II-GRS Instrument for the assessment of the methodological quality and external validation of the final statements. Results: Along with the committee chairman, 22 European experts; 7 junior experts and an independent validation committee of 11 international surgeons produced 67 guideline statements for the safe progression and dissemination of laparoscopic liver surgery. Each of the statements reached at least a 95% consensus among the experts and were endorsed by the independent validation committee. Conclusion: The European Guidelines Meeting for Laparoscopic Liver Surgery has produced a set of clinical practice guidelines that have been independently validated for the safe development and progression of laparoscopic liver surgery. The Southampton Guidelines have amalgamated the available evidence and a wealth of experts’ knowledge taking in consideration the relevant stakeholders’ opinions and complying with the international methodology standards.

The adaptation of health technology assessment reports: Identification of the need for, and development of, a toolkit to aid the process

OBJECTIVES Europe has many health technology assessment (HTA) agencies, each producing their own HTA reports. Adapting HTA reports for different contexts could reduce the need for multiple reports on the same health technology with resultant saving of time and resources. This study aims to examine and understand the process of adaptation, and to develop a toolkit that would help the adaptation of reports produced by other countries. METHODS The methods used were a review of the literature; a survey of twenty-nine European HTA organizations, two rounds of a Delphi survey, a face-to-face meeting of twenty-one European network for Health Technology Assessment (EUnetHTA) representatives, iterative rounds of review, and two rounds of quality assurance testing (termed applicability testing). RESULTS Descriptions of previous examples of adaptation in the literature are sparse. Most respondents had previous experience in adapting reports, and all believed that adaptation was useful, and there was the ability to benefit from the use of a toolkit to aid in the process. EUnetHTA Partners developed and tested an adaptation toolkit. The toolkit is composed of a series of checklists and resources that identify or clarify the relevance, reliability, and transferability of data and information from existing reports. CONCLUSIONS Consensus of opinion from twenty-nine European organizations/networks has indicated that the adaptation of HTA reports would be desirable and beneficial. A toolkit was developed to help with the adaptation of HTA reports produced in other settings. This collection of resources is available for use by all HTA agencies and can be accessed at: http://www.eunethta.net/upload/WP5/EUnetHTA_HTA_Adaptation_Toolkit_October08.pdf.

An economic evaluation of screening 60- to 70-year-old adults for hearing loss

BACKGROUND Hearing loss is common among older adults and has consequences for sufferers, families and society, but there is substantial unmet need for intervention. Screening could expedite intervention and improve outcomes. METHODS We use Markov models to estimate the incremental cost-effectiveness ratio (ICER) of potential screening programmes compared with current provision (GP-referral), from a health service perspective. Alternative options are investigated through scenario analysis. One-way and probabilistic sensitivity analyses are undertaken. RESULTS All modelled screens are cost-effective and reduce unmet need for hearing aids. The most cost-effective option identified is a one-stage audiometric screen for bilateral hearing loss ≥30 dB hearing level (HL) at age 60, repeated at ages 65 and 70. This option has an ICER of £1461 compared to GP-referral and would mean an additional 15 437 adults benefiting from hearing intervention per 100 000 population aged 60. The cost-effectiveness acceptability curve shows that screening is more cost-effective than GP-referral provided a Quality Adjusted Life Year is valued at £2000 or more. CONCLUSIONS Adult hearing screening would provide a cost-effective way to improve quality of life for older adults. We recommend piloting an audiometric screen offered to all adults age 60, 65 and 70 years to identify bilateral hearing loss of at least 30 dB HL.

Does quality of life improve in octogenarians following cardiac surgery? A systematic review

Objectives Current outcome measures in cardiac surgery are largely described in terms of mortality. Given the changing demographic profiles and increasingly aged populations referred for cardiac surgery this may not be the most appropriate measure. Postoperative quality of life is an outcome of importance to all ages, but perhaps particularly so for those whose absolute life expectancy is limited by virtue of age. We undertook a systematic review of the literature to clarify and summarise the existing evidence regarding postoperative quality of life of older people following cardiac surgery. For the purpose of this review we defined our population as people aged 80 years of age or over. Methods A systematic review of MEDLINE, EMBASE, Cochrane Library, trial registers and conference abstracts was undertaken to identify studies addressing quality of life following cardiac surgery in patients 80 or over. Results Forty-four studies were identified that addressed this topic, of these nine were prospective therefore overall conclusions are drawn from largely retrospective observational studies. No randomised controlled data were identified. Conclusions Overall there appears to be an improvement in quality of life in the majority of elderly patients following cardiac surgery, however there was a minority in whom quality of life declined (8–19%). There is an urgent need to validate these data and if correct to develop a robust prediction tool to identify these patients before surgery. Such a tool could guide informed consent, policy development and resource allocation.

Impact of NIHR HTA Programme funded research on NICE clinical guidelines: a retrospective cohort

BackgroundIt is vitally important that there is a connection between health research and clinical practice. Indications as to the impact of the research on evidence-based practice and policy can be obtained by tracking the use of outputs of health research, especially its use in clinical guidelines (CGs). This study aims to assess the proportion of National Institute for Health and Care Excellence (NICE) CGs citing National Institute for Health Research Health Technology Assessment (NIHR HTA) studies and the impact of evidence from those studies on the included NICE CGs.MethodsThis is a retrospective cohort study assessing the proportion of NICE CGs from all NICE CGs issued between April 2001 and April 2012, which cited evidence from studies funded by the NIHR HTA Programme and the impact of those studies on the CGs as the primary and secondary outcome measures.ResultsOf the cohort of NICE CGs (n = 122), 3 (2%) CGs were based on previous NIHR HTA reports and would not have been issued in that form without those NIHR HTA studies, 90 (74%) included evidence from NIHR HTA studies, and 29 (24%) did not include evidence from NIHR HTA studies. The impact of NIHR HTA evidence on NICE CGs varied in the type and quantity of data used.ConclusionsFindings suggest that NIHR HTA funded research impacts on clinical guidance from NICE and hence is well connected to both clinical practice and policy.