Randomized controlled trials do not reflect reality: Real-world analyses are critical for treatment guidelines!

Grapow, Martin; Wattenwyl, Robert von; Güller, Ulrich; Beyersdorf, Friedhelm; Zerkowski, Hans‐Reinhard

doi:10.1016/j.jtcvs.2006.03.035

Cited by 47 publications

(33 citation statements)

References 13 publications

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“…Although this is unlikely to be feasible, it may be that randomised controlled trial (RCT) methods are not the best design to test this sort of pragmatic intervention. Although traditionally viewed as the 'gold standard' design, RCTs have come under increasing criticism for their lack of clinical utility and inability to reflect real-world effectiveness (Grapow et al, 2006, Hodgson et al, 2007. Therefore, further exploration of the exercise buddy model using a more 'pragmatic trial approach' may be of most benefit (Patsopoulos, 2011).…”

Section: Discussionmentioning

confidence: 99%

Augmenting home exercise programmes in multiple sclerosis with ‘exercise buddies’: A pilot study

Toomey

Coote

2017

International Journal of Therapy and Rehabilitation

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Section: Discussionmentioning

confidence: 99%

Augmenting home exercise programmes in multiple sclerosis with ‘exercise buddies’: A pilot study

Toomey

Coote

2017

International Journal of Therapy and Rehabilitation

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“…Même si la « moyenne » peut fournir une indication intéressante, pour le malade pris en charge, les deux seuls chiffres à considérer sont donc 0 ou 100 %. Globalement, les ECR sont plus aptes à indiquer l'efficacité d'un traitement que la personne qui doit en bénéficier [5]. Le « meilleur » traitement pour un groupe de malades n'est pas obligatoirement le « meilleur » traitement pour un malade donné.…”

Section: Spécificités De La Preuve En Nutrition Cliniqueunclassified

“…De nombreux travaux méthodologiques ont mis à mal la suprématie des ECR au cours des dernières années [5]. Le Lorier et al, dans une étude parue dans le New England Journal of Medicine en 1997, avaient montré que des ECR de grande taille avaient des résultats différents de ceux des méta-analyses antérieures sur le même sujet [6].…”

Section: Introductionunclassified

Faut-il des preuves pour nourrir ?

Lerebours

2011

Réanimation

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Se poser la question, « faut-il des preuves pour nourrir ? », revient à aborder plusieurs problématiques intimement liées dans la pratique quotidienne : quelle est la nature de la preuve en médecine ? Quelles sont les spécifi-cités de la preuve en nutrition clinique ? La gestion de la preuve et de l'incertitude et ses implications éthiques. La recherche de la preuve en médecine est un problème très ancien qui a connu un regain d'intérêt important dans les années 1980 avec le développement de l'evidence-based medicine (EBM) et la place majeure, voire excessive, faite aux résultats des essais contrôlés randomisés. En dehors de quelques situations cliniques particulières et peu nombreuses, les niveaux de preuve en nutrition clinique sont faibles. Deux exemples permettent d'illustrer cette situation et d'en comprendre les raisons : le contrôle glycémique en réanimation et la nutrition entérale (NE) précoce chez le malade agressé. Les discordances entre les études peuvent s'expliquer par des différences méthodologiques, mais de manière plus profonde, elles démontrent que, contrairement à ce qu'avaient imaginé les promoteurs de l'EBM, la preuve a deux composantes, une composante factuelle et une composante contextuelle. Les faits ne naissent pas seuls, un fait scientifique ne peut pas être isolé de son contexte de production. Se poser la question « faut-il des preuves pour nourrir ? » renvoie enfin aux spécificités de la nutrition par rapport aux autres prises en charge. La nutrition n'est pas dans l'imaginaire collectif un soin courant, car la privation de nourriture est associée à la mort. L'instauration, la poursuite ou l'arrêt de la nutrition dans les situations de fin de vie doivent donc reposer sur une réflexion éthique. En conclusion, le médecin n'est pas confronté à l'application plus ou moins automatique de « l'évidence », mais à la gestion de l'incertitude et à la résolution des questions éthiques. Pour citer cette revue : Réanimation 20 (2011). Mots clés Nutrition · Agression · Éthique · Médecine par les preuvesAbstract Asking the question "do we need evidence for feeding?" raises several concerns that are closely linked in clinical practice. What is the nature of the evidence in medicine? What are the specificities of the evidence in clinical nutrition, management of the evidence and of the uncertainty, as well as ethical implications? Searching for the evidence is an old issue that became more relevant in the eighties with the development of the evidence-based medicine (EBM) theory and the increasing importance of randomised controlled trials. Except some specific clinical situations, the level of proof is often weak in clinical nutrition. Two examples illustrate this situation: the glycemic control and the early enteral nutrition in critically ill patients. Some methodological issues may explain the discrepancies between the studies, but more deeply, these discrepancies demonstrate that, in opposition with the view of EBM promoters, the proof has always two components: a factual and a contextual one. Fact...

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“…Many RCTs have been criticized as providing too little long-term follow-up, overly strict inclusion/exclusion criteria, too homogeneous a population for broad applicability, and treatment protocols that do not reflect routine clinical practice in community settings. [5] That is, these RCTs do not provide 'real-world' evidence of value that may be used for clinical decision making across heterogeneous populations [6] and that may be broadly applied to CER. Nevertheless, abandoning RCTs would be imprudent.…”

Section: Benefits Of Randomization and Limitations Of The Current CLImentioning

confidence: 99%

Generating Evidence for Comparative Effectiveness Research Using More Pragmatic Randomized Controlled Trials

et al. 2010

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Comparative effectiveness research (CER), or research design to meet the needs of post-regulatory decision makers, has been brought into the spotlight with the introduction of the American Recovery and Reinvestment Act, which provided $US1.1 billion over 2 years to support CER. In the short run, the majority of this money will be invested in observational studies and building of infrastructure; however, in the long run, we will likely see an increase in the number of randomized controlled trials (RCTs), as this method is arguably the most unbiased approach for establishing causal effect between treatments and health outcomes. RCTs are an integral component of CER for generating credible evidence on the relative value of alternative interventions in order to meet the needs of post-regulatory decision makers (patients, physicians, payers and policy makers). Explanatory phase III RCTs are fit for purpose; researchers make use of guidance documents produced by the US FDA to inform the design of these clinical trials. Historically, without explicit FDA guidance, broad patient populations, including women and minorities, often were not considered in trial design. In addition, attempts to minimize cost and maximize efficiency have led to smaller sample sizes, as is clear from the increase in 'creeping phase II-ism'. To demonstrate effectiveness, RCTs must be reflective of how an intervention will be used in the healthcare market. The concept of pragmatic clinical trials has emerged to describe those trials that are designed explicitly with this need in mind. Use of pragmatic trials will be most impactful if post-regulatory decision makers are engaged in the development of recommendations for trial design features, such as indicating outcomes measures and articulating patient populations of interest, which clearly express their evidence needs.

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Randomized controlled trials do not reflect reality: Real-world analyses are critical for treatment guidelines!

Cited by 47 publications

References 13 publications

Augmenting home exercise programmes in multiple sclerosis with ‘exercise buddies’: A pilot study

Augmenting home exercise programmes in multiple sclerosis with ‘exercise buddies’: A pilot study

Faut-il des preuves pour nourrir ?

Generating Evidence for Comparative Effectiveness Research Using More Pragmatic Randomized Controlled Trials

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