Use Of Randomized Control Trials Research Articles

Exposure to Meta-Analysis During Graduate and Postgraduate Trainings Predicts the Good Knowledge and Use of Randomized Controlled Trials, Systematic Reviews, and Meta-Analyses Among Nigerian Physiotherapists.

Background Evidence-based practice (EBP) is essential for physiotherapy as an integral part of the multidisciplinary rehabilitation team. Randomized controlled trials (RCTs), systematic reviews, and meta-analyses are the gold standard in the hierarchy of evidence. However, the extent of knowledge, attitudes, and professional use of RCTs and meta-analyses among physiotherapists in Nigeria remains unclear. Therefore, this study aimed to describe and explore the predictors of Nigerian physiotherapists' knowledge, attitudes, and professional behaviors toward RCTs, systematic reviews, and meta-analyses. Methods In this observational study, an electronic version of an adapted questionnaire assessing the knowledge, attitudes, and professional use of RCTs and meta-analyses was shared across electronic platforms of Nigerian physiotherapy professional organizations. Results We found good overall knowledge (76 {80.8%}) and attitude (83 {88.3%}) towardthe use of RCTs, systematic reviews, and meta-analyses for evaluating health interventions. Exposure to meta-analysis during graduate and postgraduate training (odds ratio {OR}, 7.102; 95% CI, 1.680-30.021; p = 0.008) and the presence of a medical library at the workplace (OR, 0.264; 95% CI, 0.070-0.997; p = 0.049) were significant predictors of good knowledge of RCTs, systematic reviews, and meta-analyses. Self-rated (OR, 56.476; 95% CI, 1.356-2357.430; p = 0.034) and overall levels of knowledge (OR, 0.013; 95% CI, 0.000-0.371; p = 0.011) predicted the good use of RCTs, systematic reviews, and meta-analyses among respondents. Discussion To equip physiotherapy practitioners with the requisite skill in using RCTs, systematic reviews, and meta-analyses, graduate and postgraduate trainings should prioritize education on the use of RCTs, systematic reviews, and meta-analyses to inform clinical decisions and practice, while capable workplaces may set up medical libraries to ease access and enhance the use of RCTs, systematic reviews, and meta-analyses.

Precision oncology medicines and the need for real world evidence acceptance in health technology assessment: Importance of patient involvement in sustainable healthcare

Precision oncology has made remarkable strides in improving clinical outcomes, offering hope to patients with historically difficult-to-treat, as well as rare or neglected cancers. However, despite rapid advancement, precision oncology has reached a critical juncture, where patient access to these life-saving medicines may be hampered by strict requirements by Health Technology Assessment (HTA) bodies for randomised controlled trials (RCTs) for assessing new medicines against appropriate comparator. The very nature of precision oncology—matching a tumour’s unique molecular alterations to targeted therapies predicted to elicit response—can make the use of RCTs very difficult, as only a very small number of patients might qualify for a given therapy within a traditional clinical trial setting. Real-world evidence (RWE) has been accepted for regulatory decision-making but has yet to reach widespread acceptance by HTA bodies. As the oncology treatment landscape has evolved towards favouring the concept of precision oncology, there is a growing need for flexibility in the way HTA bodies evaluate new medicines. We must acknowledge that current assessment methodologies can limit access to life-changing medicines for many patients who have no alternative options and that a growing number of precision oncology medicines with proven clinical benefits in rare tumours cannot be reasonably evaluated using traditional methodologies. The objectives of this paper are to advocate a change in mindset regarding best practices in drug assessment models and to propose alternative approaches when considering indications for which RWE is the most compelling data source available.

Prevention of human exposure to livestock faecal waste in the household: a scoping study of interventions conducted in sub-Saharan Africa

BackgroundPoorly managed animal faecal waste can result in detrimental environmental and public health implications. Limiting human exposure to animal waste through Animal inclusive Water Sanitation and Hygiene (A-WASH) strategies is imperative to improve public health in livestock keeping households but has received little attention to date. A small number of A-WASH interventions have previously been identified through a systematic review by another research team, and published in 2017. To inform intervention design with the most up-to-date information, a scoping study was conducted to map the existing evidence for A-WASH in sub-Saharan Africa (SSA) emerging since the previous review.MethodsThis review followed PRISMA guidelines to identify interventions in SSA published between January 2016 to October 2022. Databases searched included PubMed, PMC Europe, CabDirect and Web of Science. Studies were eligible for inclusion if they were written in English and documented interventions limiting human contact with animal faecal material in the SSA context. Key data extracted included: the intervention itself, its target population, cost, measure of effectiveness, quantification of effect, assessment of success, acceptability and limitations. These data were synthesized into a narrative, structured around the intervention type.FindingsEight eligible articles were identified. Interventions to reduce human exposure to animal faecal matter were conducted in combination with ‘standard’ human-centric WASH practices. Identified interventions included the management of human-animal co-habitation, educational programs and the creation of child-safe spaces. No novel A-WASH interventions were identified in this review, beyond those identified by the review in 2017. Randomised Controlled Trials (RCTs) were used to evaluate six of the eight identified interventions, but as effect was evaluated through various measures, the ability to formally compare efficacy of interventions is lacking.ConclusionThis study indicates that the number of A-WASH studies in SSA is increasing and the use of RCTs suggests a strong desire to create high-quality evidence within this field. There is a need for standardisation of effect measures to enable meta-analyses to be conducted to better understand intervention effectiveness. Evaluation of scalability and sustainability of interventions is still lacking in A – WASH research.

Whither ethics in research? Randomized controlled trials at the nexus of technocratization and Eurocentrism

AbstractThe use of randomized controlled trials (RCTs) as a methodological approach has become ubiquitous across disciplines, particularly in development economics. Building on the existing scholarship critiquing the widespread deployment of RCTs, this paper argues that the lack of a nuanced engagement with the concept of objectivity creates fertile grounds for the technocratization of social science research. The technocratic discourse that shapes the use of RCTs makes them susceptible to two potential dangers: perpetuating politics of partner organizations and Eurocentrism. To illustrate these issues, this paper utilizes a recently published RCT titled ‘Randomizing Religion’ as a case study. Two important conclusions are drawn by analyzing the case. First, a lack of engagement with the worldviews and assumptions from which causal mechanisms are drawn leads to a normative imposition of a Eurocentric social reality onto the marginalized communities in the Global South. Second, participation in the activities of a partner organization during the conduct of the RCT requires researchers to reflect on the organization's politics and its ramifications for marginalized communities. Finally, this paper contributes to an ongoing conversation about including strong reflexivity in the conduct of research in economics.

What is the standard of care in experimental development economics?

A central feature of experimental development economics is the use of randomized controlled trials (RCTs) to evaluate the effectiveness of prospective socioeconomic interventions. The use of RCTs in development economics raises a host of ethical issues which are just beginning to be explored. In this article, I address one ethical issue in particular: the routine use of the status quo as a control when designing and conducting a development RCT. Drawing on the literature on the principle of standard care in medical research ethics, as well as considerations of distributive justice in non-ideal circumstances, I argue that the practice of using the status quo as a control is ethically justifiable. However, I add an important qualification based on the natural duty of rescue to address the concern that my account is overly permissive.

Mechanistic evidence and exercise interventions: Causal claims, extrapolation, and implementation.

Exercise interventions and policies are widely prescribed in both sport and healthcare. Research investigating exercise interventions and policies is generally conducted using an Evidence-Based framework, placing an emphasis on evidence gathered from randomised controlled trials (RCTs). To explore the idea that, in addition to the assessment of evidence from RCTs when investigating exercise interventions, mechanistic studies ought to also be assessed and considered. This article assesses the rationale supporting the use of RCTs as evidence for exercise interventions, and the use of evidence of mechanisms in establishing efficacy, determining external validity, and tailoring interventions. The article argues that evidence from mechanistic studies ought to be considered alongside evidence from RCTs because: as RCTs investigating exercise interventions tend to be of low quality, mechanistic studies ought to be used to reinforce the evidence base; further, evidence from mechanistic studies is highly useful for both questions of extrapolation and implementation. This article argues for this on theoretical grounds, and also draws on a number of case studies.

PP205 The Use Of Real-World Evidence To Support National Institute For Health And Care Excellence Medical Technology Submissions

IntroductionAlthough randomized controlled trials (RCTs) are recognized as providing the highest level of clinical evidence, few medical device RCTs are available due to underfunding or inherent challenges associated with trial design. This study examines the extent to which real-world evidence (RWE) supports the recommendations made by the National Institute for Health and Care Excellence Medical Technologies Evaluation Programme (MTEP).MethodsAll MTEP guidance documents published online prior to October 2020 were reviewed. The “case for adoption” recommendation, type of clinical data, and clinical critiques for each MTEP submission were extracted and categorized. RWE was defined as studies with neither blinding nor prospective selection or control of patient characteristics.ResultsOf the MTEP submissions reviewed, 34 of 45 (76%) received a positive recommendation. Independent of outcome, all submissions included RWE, but only 19 (42%) utilized RCT evidence (15 were recommended and four were not). Meta-analyses of RWE were used whenever possible. The most common clinical critiques in unsuccessful submissions were the following: (i) not generalizable to the United Kingdom National Health Service (NHS); (ii) low quality; (iii) likelihood of bias; (iv) trial design faults; (v) uncertain benefit; and (vi) evidence unrelated to scope.ConclusionsThis study suggests that while the use of RCTs has not always led to a positive recommendation, RWE can be valuable in decision-making. Evidence that is generalizable to the NHS, is related to the scope, and shows clear indication of benefit is more likely to positively influence MTEP decision-making.

Outcomes reported in randomised controlled trials of major depressive disorder in older adults: protocol for a methodological review

IntroductionMajor depressive disorder (MDD or depression) is prevalent among adults aged 65 years and older. The effectiveness and safety of interventions used to treat depression is often assessed through randomised...

Randomized Controlled Trials versus Single Cohort Studies in Relapse Refractory Multiple Myeloma

Background: The Randomized Controlled Trial (RCT) is considered the gold-standard for the evaluation of treatment efficacy. For rare or end stage cancers for which there are no effective treatments, or the number of patients is sparse, the use of RCTs for the assessment of efficacy and safety may be difficult. In these circumstances the single cohort study (SC) can be considered as an alternative to the RCT. Purpose: The purpose of this study was to compare the measures of efficacy as assessed with estimates of the Overall Response Rate (ORR) or Overall Survival (OS) obtained in RCT and SC studies that evaluate the same treatments for Relapse Refractory Multiple Myeloma (RRMM). The study also compared the estimates of ORR and OS ratios between treatments estimated in RCTs and extrapolated in SCs for the same treatments of RRMM. Methods: The study utilized data from 42 RCTs and 47 SCs assessing 18 different treatment protocols for RRMM that were identified through a MEDLINE search. Results: The results showed that there were no material differences in the demographics of patients enrolled in RCTs and SCs. The estimates of ORR and OS obtained in RCTs and SCs were comparable. Statistically significant Intra-Class Correlation Coefficient (ICC = 0.618, P = 0.027) was observed for ORR and for OS (ICC = 0.734; P = 0.014) indicating good agreement between RCTs and SCs. Treatment effect size as measured by the ORR and OS ratios (new treatment / control) was ascertained directly from RCTs and extrapolated for SCs based on the control ORR and OS observed in RCTs. There was agreement between RCTs and SCs with respect to the magnitude and direction of the ORR ratios (91% of the studies) and the OS ratios (75% of the studies). With respect to the conclusion regarding the relative efficacy of the new treatment versus the control, there was agreement between RCTs and SCs for 8/11 treatments based on ORR ratios and for 6 / 8 based on OS ratios. Conclusions: The results of this study have shown that for RRMM single cohort studies can be used to assess the efficacy of new treatments, given that sufficient data are available on controls treatments used as standard of care. The results may have implications for the evaluation of treatments of rare and advanced cancers as well as other conditions.

Evidence-based policies, nudge theory and Nancy Cartwright: a search for causal principles

AbstractNancy Cartwright argues that evidence-based policies should not only rely on randomized controlled trials (RCTs) to test their effectiveness – they should also use horizontal and vertical searches to find support factors and causal principles that help define how those policies work. This paper aims at analyzing Cartwright's epistemology regarding evidence-based policies and their use of RCTs while applying her findings to current research involving nudges as behavioral public policy interventions. Holding a narrowly instrumental view of rationality, nudge theory tends to neglect other expressive components. Policymakers, in their quest for causal principles, should consider the expressive rationality of individuals in their research. This inclusion would not only increase the effectiveness of nudges, but also address some ethical issues related to people's autonomy when targeted by these interventions.

Gli esperimenti nelle scienze sociali: molta strada resta da fare per un dibattito maturo sul tema

Randomized Controlled Trial (RCT) is a power tool to assess causality. The use of RTCs among sociologists is limited by common constraints, for example the ethical issues and the non-manipulability of several individual characteristics (i.e. social origins, education, gender, etc.). Moreover, limited use is due also to the lack of knowledge and misunderstanding about how RCTs can generate robust causal inference. In this essay we confront these topics, moving from a recent Italian book about experiments (Rago, 2018). Focusing on several objections to the internal and external validity of RCTs, we show a small amount of the large body of methodological solutions provided by consolidated international literature in the field. In doing so, we try to eliminate the most common misunderstandings related to RCTs. We also argue that a larger use of RCTs may be useful for sociology, thanks to the possibility to integrate rigorous causal estimates with our deeper knowledge of mechanisms underlying social phenomena.

What role should randomized control trials play in providing the evidence base for conservation?

AbstractThe effectiveness of many widely used conservation interventions is poorly understood because of a lack of high-quality impact evaluations. Randomized control trials (RCTs), in which experimental units are randomly allocated to treatment or control groups, offer an intuitive way to calculate the impact of an intervention by establishing a reliable counterfactual scenario. As many conservation interventions depend on changing people's behaviour, conservation impact evaluation can learn a great deal from RCTs in fields such as development economics, where RCTs have become widely used but are controversial. We build on relevant literature from other fields to discuss how RCTs, despite their potential, are just one of a number of ways to evaluate impact, are not feasible in all circumstances, and how factors such as spillover between units and behavioural effects must be considered in their design. We offer guidance and a set of criteria for deciding when RCTs may be an appropriate approach for evaluating conservation interventions, and factors to consider to ensure an RCT is of high quality. We illustrate this with examples from one of the few concluded RCTs of a large-scale conservation intervention: an incentive-based conservation programme in the Bolivian Andes. We argue that conservation should aim to avoid a rerun of the polarized debate surrounding the use of RCTs in other fields. Randomized control trials will not be feasible or appropriate in many circumstances, but if used carefully they can be useful and could become a more widely used tool for the evaluation of conservation impact.

Evidence to Support Inclusion of Pharmacogenetic Biomarkers in Randomised Controlled Trials.

Pharmacogenetics and biomarkers are becoming normalised as important technologies to improve drug efficacy rates, reduce the incidence of adverse drug reactions, and make informed choices for targeted therapies. However, their wider clinical implementation has been limited by a lack of robust evidence. Suitable evidence is required before a biomarker’s clinical use, and also before its use in a clinical trial. We have undertaken a review of five pharmacogenetic biomarker-guided randomised controlled trials (RCTs) and evaluated the evidence used by these trials to justify biomarker inclusion. We assessed and quantified the evidence cited in published rationale papers, or where these were not available, obtained protocols from trial authors. Very different levels of evidence were provided by the trials. We used these observations to write recommendations for future justifications of biomarker use in RCTs and encourage regulatory authorities to write clear guidelines.

The trials of evidence-based practice in education: a systematic review of randomised controlled trials in education research 1980–2016

ABSTRACTBackground: The use of randomised controlled trials (RCTs) in education has increased significantly over the last 15 years. However, their use has also been subject to sustained and rather trenchant criticism from significant sections of the education research community. Key criticisms have included the claims that: it is not possible to undertake RCTs in education; RCTs are blunt research designs that ignore context and experience; RCTs tend to generate simplistic universal laws of ‘cause and effect’; and that they are inherently descriptive and contribute little to theory.Purpose: This article seeks to assess the above four criticisms of RCTs by considering the actual evidence in relation to the use of RCTs in education in practice.Design and methods: The article is based upon a systematic review that has sought to identify and describe all RCTs conducted in educational settings and including a focus on educational outcomes between 1980 and 2016. The search is limited to articles and reports published in English.Results: The systematic review found a total of 1017 unique RCTs that have been completed and reported between 1980 and 2016. Just over three quarters of these have been produced over the last 10 years, reflecting the significant increase in the use of RCTs in recent years. Overall, just over half of all RCTs identified were conducted in North America and a little under a third in Europe. The RCTs cover a wide range of educational settings and focus on an equally wide range of educational interventions and outcomes. The findings not only disprove the claim that it is not possible to do RCTs in education but also provide some supporting evidence to challenge the other three key criticisms outlined earlier.Conclusions: While providing evidence to counter the four criticisms outlined earlier, the article suggests that there remains significant progress to be made. The article concludes by outlining some key challenges for researchers undertaking RCTs in education.

Why are there (almost) no randomised controlled trial-based evaluations of business support programmes?

Based on the achievements of randomised controlled trials (RCTs) in medicine, and the need for effective government interventions in support of business, some have advocated for the use of RCTs in the evaluation of business support programmes (BSPs). Notwithstanding these recommendations, the use of RCTs in the evaluation of BSPs has been resisted by (almost) all. Policy makers and managers are correct in their reluctance to undertake RCT-based evaluations for four reasons. First, while RCTs require the random allocation of support, judicious programmes select firms on the basis of potential and amenability to support. Second, while RCTs require treatments that exhibit low variability, the most effective BSPs draw upon substantive knowledge to provide support that is customised. Third, BSPs aim to produce outliers—firms whose performance is exceptional. When outliers are present, very large samples will be required to produce reliable results. Finally, an RCT may not yield a meaningful contribution to knowledge. The strength of an RCT is its ability to estimate the magnitude of the treatment effect under controlled conditions. But where much depends on the nature of participants and circumstances, we seek evidence of what works, for whom, in which circumstances, and why.

Informing efficient randomised controlled trials: exploration of challenges in developing progression criteria for internal pilot studies

ObjectivesDesigning studies with an internal pilot phase may optimise the use of pilot work to inform more efficient randomised controlled trials (RCTs). Careful selection of preagreed decision or ‘progression’ criteria...

Notes on the use of randomised controlled trials to evaluate complex interventions: Community treatment orders as an illustrative case.

Over the past seven decades, randomised controlled trials (RCTs) have revolutionised clinical research and achieved a gold standard status. However, extending their use to evaluate complex interventions is problematic. In this paper we will demonstrate that complex intervention RCTs violate the necessary premises that govern the RCTs logic and underpin their rigour. The lack of blinding, heterogeneity of participants, as well as poor treatment standardisation and difficulty of controlling for confounders, which characterise complex intervention RCTs, can potentially be profoundly detrimental to their integrity. Proponents of this approach argue that matching "real world" circumstances, while maintaining the randomised design, enhances external validity. We counter this argument by pointing out that an inverted U relation exists between internal and external validity, and thus relaxing the experimental conditions beyond a certain threshold can potentially paradoxically render the RCT externally invalid, i.e. its results cannot be used anywhere. We shall illustrate the inappropriate use of RCTs to evaluate community treatment orders and propose an alternative epistemic model that is based on mechanistic reasoning and Cartwright's capacity concept.

On the Randomisation Gallery:Random Reflections on a Popular Method in Medical Research

Objective: Randomized controlled trials (RCTs) and meta-analysis are increasingly utilized methods in medicine. There is, however, substantial critique on both from the theoretical as well as from the practical side. Methods: Seven points on RCTs and three on meta-analysis were choosen to summarize the current critique. Studies critically reviewing the quality of meta-analyses in various fields of medicine were narratively reviewed. Results: The typical RCT is short, performed on a small group of selected patients without severe comorbidity. It is expensive and favors drugs over other forms of therapies. Furthermore, neither patients nor doctors much like RCTs, they feel restricted in their choice of treatments. Finally, it can be problematic to obtain informed consent in disorders affecting decision making, e.g. acute psychosis severe depression or dementia. The evaluation of existing meta-analyses varied between acceptable and poor, good judgments were not often found. Conclusion: The current uncritical use of RCTs and meta-analyses in medicine is not appropriate. Doctors using meta-analyses simply thinking to gather information efficiently are often unaware that there is evidence for bias. Keywords: Randomized controlled trials; Meta-analysis; Quality assessment

Randomised Controlled Trials in WOHP Interventions: A Review and Guidelines for Use

Randomised controlled trials (RCTs) have long been considered a gold standard for intervention design and the most rigorous method for understanding causal mechanisms. However, their implementation in work and organisational health psychology (WOHP) can be challenging. We review the use of RCTs in WOHP interventions and demonstrate that their adoption has been relatively scarce in comparison to areas such as health psychology and medical sciences. For WOHP researchers to be able to compare the effectiveness of their work‐specific health interventions with other interventions in health and medicine, it is important that the design methodology, rigor, and reporting are comparable. Thus, there is a need for a clearer road map and guidance for WOHP researchers, to encourage greater use of RCTs in WOHP intervention research. In the paper, we provide an overview of RCTs, and review past research that has utilised an RCT design when evaluating WOHP interventions. We develop an adapted RCT checklist for use in WOHP settings, which takes specific organisational issues into account. Thus, our paper provides for future researchers a clearer road map for the design and reporting of WOHP RCT studies.

Meeting the Challenges of Intervention Research in Health Science: An Argument for a Multimethod Research Approach.

Research within health science is often based on developing, implementing and evaluating interventions in a randomized controlled trial (RCT) design, with patients or other health care users as the target group. The results of RCTs can have limited generalizability. Since a trial often takes place in a controlled setting, it may be difficult to implement the results in other settings. Successful implementation in practice requires knowledge of the context and the social mechanisms and processes through which an intervention works. It is therefore important to secure such knowledge of high quality. The aim of this paper was to present and discuss how intervention research in RCT designs can be developed and strengthened by using a multimethod research approach. First, we focus on four considerations relating to the use of RCTs, namely objectivity and linearity, contextual dimensions, generalizability, and complex interventions. Second, a multimethod research approach including the terms 'research style' and 'forms of integration' is presented to address the four considerations. Third, a Danish intervention study is presented in order to discuss the potential of this multimethod research approach. We conclude by suggesting that future intervention studies should consider the potential for combining different research styles and forms of integration to the benefits of the patients and other health care users as the target group.