scholarly journals Can we rely on non-randomised studies? Findings from a meta-epidemiological review

2019 ◽  
Vol 29 (Supplement_4) ◽  
Author(s):  
J C Rejon-Parrilla ◽  
M Salcher-Konrad ◽  
M Nguyen ◽  
K Davis ◽  
P Jonsson ◽  
...  
Keyword(s):  
Health Economic ◽  
Analytical Methods ◽  
Large Scale ◽  
Treatment Effects ◽  
Controlled Trial ◽  
Health Economic Evaluation ◽  
Risk Of Bias ◽  
Economic Decision Making ◽  
Wide Range ◽  
Randomised Controlled

Abstract Background Increasingly, health technology assessment (HTA) agencies must decide whether new medicines should be used routinely in the absence of randomised controlled trial (RCT) data, relying solely on non-randomised studies (NRS), which are at high risk of bias due to confounding. Against the background of increased availability and improved methods to analyse non-randomised data (e.g., propensity score methods and instrumental variables), it is important for decision-makers to have guidance on the analysis and interpretation of NRS to inform health economic evaluation. We therefore aimed to systematically and empirically assess the performance of NRS using different analytical methods as compared to RCTs and develop recommendations on the basis of our findings. Methods We conducted a large-scale meta-epidemiological review to obtain estimates of the discrepancy in treatment effects in matched RCTs and NRS of pharmacologic interventions from published meta-analyses indexed in MEDLINE and the Cochrane Database of Systematic Reviews. We also consulted with HTA bodies, regulators and academics from five European countries to learn from their experience with using non-randomised evidence. Results We compiled the largest dataset of clinical topics with matching RCTs and NRS using various analytical methods to date, covering >100 unique clinical questions. Incorporating information on direction of effect and effect size from >700 unique studies, the dataset can be used to evaluate discrepancies in treatment effects between study designs across a wide range of therapeutic areas. Conclusions An empirically based understanding of the risk of bias in NRS is required in order to promote the adequate use of non-randomised evidence as input for health economic decision-making.

scholarly journals Efficacy and cost-effectiveness of therapist-guided internet-delivered behaviour therapy for children and adolescents with Tourette syndrome: study protocol for a single-blind randomised controlled trial

2021 ◽  
Author(s):  
Per Andrén ◽  
Lorena Fernández de la Cruz ◽  
Kayoko Isomura ◽  
Fabian Lenhard ◽  
Charlotte L Hall ◽  
...  
Keyword(s):  
Health Economic ◽  
Primary Endpoint ◽  
Controlled Trial ◽  
Health Economic Evaluation ◽  
Primary Objective ◽  
Behaviour Therapy ◽  
Tic Severity ◽  
Randomised Controlled ◽  
Single Blind

Background: Treatment guidelines recommend behaviour therapy (BT) for patients with Tourette syndrome (TS) and chronic tic disorder (CTD). However, BT is rarely accessible due to limited availability of trained therapists and long travel distances to specialist clinics. Internet-delivered BT has the potential of overcoming these barriers through remote delivery of treatment with minimal therapist support. In the current protocol, we outline the design and methods of a randomised controlled trial (RCT) evaluating an internet-delivered BT programme referred to as BIP TIC. The trial’s primary objective is to determine the clinical efficacy of BIP TIC for reducing tic severity in young people with TS/CTD, compared with an active control intervention. Secondary objectives are to investigate the 12-month durability of the treatment effects and to perform a health economic evaluation of the intervention.Methods: In this single-blind superiority RCT, 220 participants (9-17 years) with TS/CTD throughout Sweden will be randomised to 10-12 weeks of either therapist-supported internet-delivered BT based on exposure with response prevention (BIP TIC) or therapist-supported internet-delivered education. Data will be collected at baseline, 3 and 5 weeks into the treatment, at post-treatment, and 3, 6, and 12 months post-treatment. The primary endpoint is the 3-month follow-up. The primary outcome is tic severity as measured by the Yale Global Tic Severity Scale – Total tic severity score. Treatment response is operationalised as scores of “Very much improved” or “Much improved” on the Clinical Global Impression – Improvement scale, administered at the primary endpoint. Outcome assessors will be blind to treatment condition at all assessment points. A health economic evaluation of BIP TIC will be performed, both in the short term (primary endpoint) and the long term (12-month follow-up). There are no planned interim analyses. Discussion: Participant recruitment started on 26 April 2019 and finished on 9 April 2021. The final participant will reach the primary endpoint in September 2021 and the 12-month follow-up in June 2022. Data analysis for the primary objective will commence after the last participant reaches the primary endpoint.

scholarly journals Effectiveness of interventions for people living with dementia and their carers in Chinese communities: protocol for a systematic review and meta-analysis of randomised controlled trials

BMJ Open ◽  
2021 ◽  
Vol 11 (8) ◽  
pp. e047560
Author(s):  
Cheng Shi ◽  
Shuangzhou Chen ◽  
Maximilian Salcher-Konrad ◽  
Jacky C P Choy ◽  
Hao Luo ◽  
...  
Keyword(s):  
Systematic Review ◽  
Controlled Trial ◽  
Meta Analysis ◽  
Risk Of Bias ◽  
Sufficient Information ◽  
Ageing Population ◽  
China National Knowledge Infrastructure ◽  
Chinese Populations ◽  
Wide Range ◽  
Randomised Controlled

IntroductionAs the largest and most rapidly ageing population, Chinese people are now the major driver of the continued growth in dementia prevalence globally. The need for evidence-based interventions in Chinese communities is urgent. Although a wide range of pharmacological and non-pharmacological interventions for dementia have been trialled in Chinese populations, the evidence has not been systematically synthesised. This systematic review and meta-analysis aims to map out the interventions for people living with dementia and their carers in Chinese communities worldwide and compare the effectiveness of these interventions.Methods and analysisThis protocol followed the Preferred Reporting Items for Systematic Review and Meta-Analysis Protocols checklist. We will search Chinese (China National Knowledge Infrastructure, WanFang DATA) and English bibliographical databases (MEDLINE, EMBASE, PsycINFO, CINAHL Plus, Global Health, WHO Global Index Medicus, Virtual Health Library, Cochrane CENTRAL, Social Care Online, BASE, MODelling Outcome and cost impacts of interventions for DEMentia (MODEM) Toolkit, Cochrane Database of Systematic Reviews), complemented by hand searching of reference lists. We will include studies evaluating the effectiveness of interventions for dementia or mild cognitive impairment in Chinese populations, using a randomised controlled trial design, and published between January 2008 and June 2020. We will use a standardised form to extract data and Version 2 of the Cochrane risk-of-bias tool for randomised trials to assess the risk of bias of the included studies. Collected data will be fully interpreted with narrative synthesis and analysed using pairwise and network meta-analyses to pool intervention effects where sufficient information is available. We will perform subgroup analysis and meta-regression to explore potential reasons for heterogeneity.Ethics and disseminationNo formal ethics approval is required for this protocol. The findings will facilitate the development of studies on interventions for dementia and timely inform dementia policymaking and practice. Planned dissemination channels include peer-reviewed publications, conference presentations, public events and websites.PROSPERO registration numberCRD42019134135.

scholarly journals Efficacy and cost-effectiveness of therapist-guided internet-delivered behaviour therapy for children and adolescents with Tourette syndrome: study protocol for a single-blind randomised controlled trial

Trials ◽  
2021 ◽  
Vol 22 (1) ◽  
Author(s):  
Per Andrén ◽  
Lorena Fernández de la Cruz ◽  
Kayoko Isomura ◽  
Fabian Lenhard ◽  
Charlotte L. Hall ◽  
...  
Keyword(s):  
Health Economic ◽  
Primary Endpoint ◽  
Controlled Trial ◽  
Health Economic Evaluation ◽  
Primary Objective ◽  
Behaviour Therapy ◽  
Tic Severity ◽  
Randomised Controlled ◽  
Single Blind

Abstract Background Treatment guidelines recommend behaviour therapy (BT) for patients with Tourette syndrome (TS) and chronic tic disorder (CTD). However, BT is rarely accessible due to limited availability of trained therapists and long travel distances to specialist clinics. Internet-delivered BT has the potential of overcoming these barriers through remote delivery of treatment with minimal therapist support. In the current protocol, we outline the design and methods of a randomised controlled trial (RCT) evaluating an internet-delivered BT programme referred to as BIP TIC. The trial’s primary objective is to determine the clinical efficacy of BIP TIC for reducing tic severity in young people with TS/CTD, compared with an active control intervention. Secondary objectives are to investigate the 12-month durability of the treatment effects and to perform a health economic evaluation of the intervention. Methods In this single-blind superiority RCT, 220 participants (9–17 years) with TS/CTD throughout Sweden will be randomised to 10–12 weeks of either therapist-supported internet-delivered BT based on exposure with response prevention (BIP TIC) or therapist-supported internet-delivered education. Data will be collected at baseline, 3 and 5 weeks into the treatment, at post-treatment, and 3, 6, and 12 months post-treatment. The primary endpoint is the 3-month follow-up. The primary outcome is tic severity as measured by the Yale Global Tic Severity Scale – Total Tic Severity Score. Treatment response is operationalised as scores of “Very much improved” or “Much improved” on the Clinical Global Impression – Improvement scale, administered at the primary endpoint. Outcome assessors will be blind to treatment condition at all assessment points. A health economic evaluation of BIP TIC will be performed, both in the short term (primary endpoint) and the long term (12-month follow-up). There are no planned interim analyses. Discussion Participant recruitment started on 26 April 2019 and finished on 9 April 2021. The total number of included participants was 221. The final participant is expected to reach the primary endpoint in September 2021 and the 12-month follow-up in June 2022. Data analysis for the primary objective will commence after the last participant reaches the primary endpoint. Trial registration ClinicalTrials.gov NCT03916055. Registered on 16 April 2019.

scholarly journals Effects of a workplace prevention programme for problem gambling: study protocol for a cluster randomised controlled trial

BMJ Open ◽  
2017 ◽  
Vol 7 (9) ◽  
pp. e015963 ◽  
Author(s):  
Jonas Rafi ◽  
Ekaterina Ivanova ◽  
Alexander Rozental ◽  
Per Carlbring
Keyword(s):  
Problem Gambling ◽  
Large Scale ◽  
Controlled Trial ◽  
Public Health Problem ◽  
Cluster Randomised Controlled Trial ◽  
Severity Index ◽  
The Body ◽  
Prevention Programme ◽  
Open Access Journals ◽  
Randomised Controlled

IntroductionDespite being considered a public health problem, no prevention programme for problem gambling in workplace settings has been scientifically evaluated. This study aims to fill a critical gap in the field of problem gambling by implementing and evaluating a large-scale prevention programme in organisations.Methods and analysisTen organisations, with a total of n=549 managers and n=8572 employees, will be randomised to either receiving a prevention programme or to a waitlist control condition. Measurements will be collected at the baseline and 3, 12 and 24 months after intervention. The primary outcome of interest is the managers’ inclination to act when worried or suspicious about an employee’s problem gambling or other harmful use. Additional outcomes of interest include the Problem Gambling Severity Index and gambling habits in both managers and employees. Furthermore, qualitative analyses of the responses from semistructured interviews with managers will be performed.Ethics and disseminationThis study has been approved by the regional ethics board of Stockholm, Sweden, and it will contribute to the body of knowledge concerning prevention of problem gambling. The findings will be published in peer-reviewed, open-access journals.Trial registration numberNCT02925286; Pre-results.

scholarly journals Better Outcomes for Older people with Spinal Trouble (BOOST) Trial: a randomised controlled trial of a combined physical and psychological intervention for older adults with neurogenic claudication, a protocol

BMJ Open ◽  
2018 ◽  
Vol 8 (10) ◽  
pp. e022205 ◽  
Author(s):  
Esther Williamson ◽  
Lesley Ward ◽  
Karan Vadher ◽  
Susan J Dutton ◽  
Ben Parker ◽  
...  
Keyword(s):  
Older Adults ◽  
Randomised Controlled Trial ◽  
Older People ◽  
Best Practice ◽  
Psychological Intervention ◽  
Controlled Trial ◽  
Health Economic Evaluation ◽  
Community Dwelling ◽  
Neurogenic Claudication ◽  
Randomised Controlled

IntroductionNeurogenic claudication due to spinal stenosis is common in older adults. The effectiveness of conservative interventions is not known. The aim of the study is to estimate the clinical and cost-effectiveness of a physiotherapist-delivered, combined physical and psychological intervention.Methods and analysisThis is a pragmatic, multicentred, randomised controlled trial. Participants are randomised to a combined physical and psychological intervention (Better Outcomes for Older people with Spinal Trouble (BOOST) programme) or best practice advice (control). Community-dwelling adults, 65 years and over, with neurogenic claudication are identified from community and secondary care services. Recruitment is supplemented using a primary care-based cohort. Participants are registered prospectively and randomised in a 2:1 ratio (intervention:control) using a web-based service to ensure allocation concealment. The target sample size is a minimum of 402. The BOOST programme consists of an individual assessment and twelve 90 min classes, including education and discussion underpinned by cognitive behavioural techniques, exercises and walking circuit. During and after the classes, participants undertake home exercises and there are two support telephone calls to promote adherence with the exercises. Best practice advice is delivered in one to three individual sessions with a physiotherapist. The primary outcome is the Oswestry Disability Index at 12 months. Secondary outcomes include the 6 Minute Walk Test, Short Physical Performance Battery, Fear Avoidance Beliefs Questionnaire and Gait Self-Efficacy Scale. Outcomes are measured at 6 and 12 months by researchers who are masked to treatment allocation. The primary statistical analysis will be by ‘intention to treat’. There is a parallel health economic evaluation and qualitative study.Ethics and disseminationEthical approval was given on 3 March 2016 (National Research Ethics Committee number: 16/LO/0349). This protocol adheres to the Standard Protocol Items: Recommendations for Interventional Trials checklist. The results will be reported at conferences and in peer-reviewed publications using the Consolidated Standards of Reporting Trials guidelines. A plain English summary will be published on the BOOST website.Trial registration numberISRCTN12698674; Pre-results.

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